News

by Jason Parker, WRAL TechWire

RALEIGH – Opus Genetics recently announced it would open its third preclinical trial, just a few months after the company formed with a $19 million seed financing round led by the venture capital arm of the Foundation Fighting Blindness, the RD Fund.

The company, founded by the RD Fund, also received seed funding from the Manning Family Foundation and Bios Partners, and is the first spin-out company that was internally conceived and launched by the RD Fund to further the mission of the foundation, which is to advance the treatments and cures for blinding retinal diseases.

WRAL TechWire interviewed the company’s acting CEO, Ben Yerxa, PhD, who also serves as CEO of the Foundation Fighting Blindness and the RD Fund, about the company’s origins, mission, and technology.  A lightly edited transcript of that interview follows.

WRAL TechWire (TW): Tell us more about how and why the company was formed?

Ben Yerxa, PhD, acting CEO of Opus Genetics (Yerxa): Opus Genetics is a gene therapy company focused on preserving or potentially improving vision for patients with inherited retinal diseases with a unique model and purpose.  Opus is uniquely positioned to bring experts, resources and patients together to efficiently advance ocular gene therapies for patients that to date have been neglected.

We’re fortunate to have our scientific founders, who have dedicated their lives to working on these diseases.  The timing, founders and financing came together at the right time to advance these therapies toward patients.

Opus scientific founders are Jean Bennett, M.D., Ph.D., the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania, and Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal Ocular Therapeutics (CAROT), and Eric Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School.

This opportunity was uniquely suited to working together—technology that is de-risked through rigorous preclinical studies in models that emulate human disease, programs hyper focused in genetic retinal disease, relationships with the best scientists and organizations, and groups that all deeply understand the patient experience and the science.

Our  adeno-associated virus (AAV) based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. The company’s lead programs will focus on treatments delivered subretinally to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA), a rare form of pediatric blindness.

TW: What is the scope of the challenge with retinal disease, and why did the founders and company choose to work to address these? 

Yerxa: There is significant unmet need in the treatment of these rare blinding conditions, and we see the opportunity for a proven, de-risked, efficient path to the clinic for these desperately needed new therapies for patients.  This team and scientists have all dedicated their careers to working to improve vision for these devastating blinding conditions.

OPGx-001 is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein.  OPGx-002 will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13).  OPGx-003 is a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene.

While LCA is a group of rare inherited retinal diseases characterized by degeneration of photoreceptors that affect about 1 in 40,000 newborns, LCA5 affects approximately one in 1.7 million people, retinal dystrophy caused by mutations in the RDH12 gene affects one in 288,000 people, and disease caused by mutations in the NMNAT1 gene affects one 432,000 people in the U.S.

Another way to think about it is in the US and EU combined these indications have hundreds to low thousands of patients.

TW: The company launched with $19 million in seed funding—how do you plan to allocate and invest this capital?

Yerxa: The $19 million in seed financing was led by the RD Fund, which also founded the company, with participation from the Manning Family Foundation and Bios Partners.

This funding provides Opus with the resources to advance our pipeline of three programs, including our first program (OPGx-001) entering the clinic in 2022.  We plan to continue pursuing additional programs and develop our manufacturing plans in parallel.

A core tenet of our approach is to build an engine to bring multiple rare genetic retinal disease therapeutics into the clinic and ultimately to the patients who need them.  We will disclose additional financing and commercialization plans at the appropriate time.

TW: The company launched in Raleigh—why?

Yerxa: The Triangle is a great place to recruit top talent, and to grow biotech companies, especially in the field of gene therapy. Additionally, the RD Fund is based here, so as its first spinout, it just made sense for Opus to be founded here and gain some early operational and resource efficiencies.

As a rapidly evolving company, we anticipate expanding as we advance our programs toward the clinic. We recently filled two cornerstone leadership roles for the company—Chief Operating Officer Joe Schachle and Chief Scientific Officer Dr. Ash Jayagopal—and we plan to hire additional talent for key roles across the organization.

The idea behind Opus is not about one program, it’s about building an engine to solve as many blinding degenerative retinal diseases as possible.  There are currently over 260 genes known to cause inherited retinal diseases, so our opportunities are substantial.  The infrastructure can be used for multiple products to efficiently move validated science and/or well characterized material by preeminent leaders in the field toward patients leveraging scalable, strategic manufacturing and processes.

We see manufacturing as the lynchpin of our success, in developing the capability and strategically managing the cadence of our clinical timelines to produce therapeutics most efficiently, leveraging our founders’ expertise and learnings over a lifetime of research and development in this area.

Because these are small populations and we treat the eye, our strategy is built around high-quality, small-scale manufacturing—it is a scale down problem, not a scale up problem.  We are working to develop our own strategy for the most efficient way to manufacture small batches using high quality GMP material.  In the meantime, we want to work with a CDMO that embraces our model and is willing to produce small batches.

TW: What have people “missed” in the last two years in gene therapy and in the life sciences?  What are the emerging or existing trends in the sector that folks ought to pay attention to in 2022 and beyond?

Yerxa: The rise of the role of venture philanthropy in driving the life sciences discovery engine, and it’s just the tip of the iceberg: we’re excited about furthering the mission of venture philanthropy in this way.  Opus is the RD Fund’s first company which we have conceived, led the financing, and launched ourselves. Patient donations fund the RD Fund so its majority stakeholders are patients themselves.

That ocular gene therapy is really leading the way in gene therapy—LUXTURNA® (Spark Therapeutics, Philadelphia) was the first approved gene therapy and created by Opus scientific founder Dr. Jean Bennett.  There are several advantages of ocular gene therapy vs. other gene therapy areas, including: small dose required; ability to precisely place the dose in the desired location (subretinally); high levels of immune privilege in the back of the eye; and ability to observe the safety and efficacy readily and non-invasively with various imaging techniques and functional assays.

TW: The company announced last week that it would begin its third preclinical trial—tell us more about all three. 

Opus’ first three programs are licensed from the institutions of its scientific founders—OPGx-001 and OPGx-002 from Penn, and OPGx-003 from Harvard MEEI (Mass Eye and Ear).

Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein.  This first clinical study will be a dose escalation study in a small number of patients in order to establish safety and potentially to obtain initial efficacy readouts.  Opus expects to file an IND for its OPGx-001 program in early 2022 and enter the clinic in mid-2022.

The company’s second program, OPGx-002, will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13).  We are currently working towards an IND in the next 12-18 months.

OPGx-003 is a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene.  Opus expects to file an IND for OPGx-003 in 2023.

We hope to announce additional programs, partnerships and hirings soon, while building out our earlier stage development and manufacturing work.  Opus is bringing together the best instruments of science—the expertise of pioneers in ocular gene therapy, patient voices, committed investors, and strong research—to create important new therapies for patients.  This will be our greatest work.

Opus Genetics announced an agreement to license its third preclinical program focused on an inherited retinal disease from Massachusetts Eye and Ear, Harvard Medical School’s ophthalmic teaching hospital, according to a press release.

The OPGx-003 program is a gene augmentation therapy designed to prevent functional degeneration in the retina of pediatric patients with NMNAT1 mutations, which cause a type of Leber congenital amaurosis, and is based on the expertise of Eric Pierce, MD, PhD, director of the Ocular Genomics Institute, professor at Massachusetts Eye and Ear and Harvard Medical School and scientific cofounder of Opus Genetics.

“I’ve seen firsthand the need for new treatments for rare inherited retinal diseases,” Pierce said in the release. “As someone who has devoted my life to research in this space, I believe strongly that Opus is an ideal company to advance this work to make a difference for patients in need.”

“We’re thrilled to collaborate with Dr. Pierce, who will bring his expertise in retinal gene therapy to researching a novel treatment of NMNAT1-associated retinal degeneration,” Ash Jayagopal, PhD, chief scientific officer of Opus Genetics, said in the release. “Adding OPGx-003 to the Opus pipeline further underscores our commitment to bringing the required resources and expertise together to take promising science from the lab through the clinic and ultimately to patients who need it, and reinforces the importance of our innovative patient-focused model.”

The company expects to apply for FDA investigational new drug status for OPGx-003 in the first half of 2023, according to the release.

Program targets NMNAT1 gene based on the work of Opus scientific founder Dr. Eric Pierce

RALEIGH, N.C., Nov. 11, 2021 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced an agreement to license its third preclinical program to address mutations in the NMNAT1 gene, which cause a specific form of Leber congenital amaurosis (LCA), from Massachusetts Eye and Ear, the primary teaching hospital at Harvard Ophthalmology.

The new program, OPGx-003, is based on the work of Eric Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and scientific co-founder of Opus. OPGx-003 is a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene. Recent preclinical data have demonstrated the potential for this gene augmentation approach to achieve stable rescue of retinal structure and function. Opus expects to file an IND for OPGx-003 in the first half of 2023.

“We’re thrilled to collaborate with Dr. Pierce, who will bring his expertise in retinal gene therapy to researching a novel treatment of NMNAT1-associated retinal degeneration,” said Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus Genetics. “Adding OPGx-003 to the Opus pipeline further underscores our commitment to bringing the required resources and expertise together to take promising science from the lab through the clinic and ultimately to patients who need it, and reinforces the importance of our innovative patient-focused model.”

“I’ve seen firsthand the need for new treatments for rare inherited retinal diseases,” said Dr. Pierce. “As someone who has devoted my life to research in this space, I believe strongly that Opus is an ideal company to advance this work to make a difference for patients in need.”

Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. The company’s second program, OPGx-002, will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13).

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com

Disclosures
Dr. Pierce holds equity in and serves as a consultant for Opus Genetics.

Media Contact:
Heather Anderson
919-827-5539
handerson@6degreespr.com

Opus Genetics Announces Presence at Eyecelerator@AAO 2021

11/08/2021 | 08:34am EST

RALEIGH, N.C., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced that Ben Yerxa, Ph.D., acting CEO of Opus and CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, will deliver a company presentation at Eyecelerator@AAO 2021, on Thursday, November 11, 2021, in New Orleans.

Dr. Yerxa’s Opus presentation will be part of the Retina Spotlight: Meet the Game-Changing Innovators With the Most Promising Strategies For Impacting Retina session, which includes a panel and a diverse line-up of five companies presenting innovations at all stages in a unique, interactive format.

“We’re thrilled to be invited to introduce Opus and highlight our innovative model at Eyecelerator,” said Dr. Yerxa. “Created by the Foundation’s RD Fund, Opus emerged from stealth mode in September singularly focused on developing and manufacturing orphaned AAV-based gene therapies to treat neglected, orphan inherited retinal diseases. We look forward to showcasing our unique, patient-guided approach at this premiere industry event.”

Eyecelerator@AAO 2021 is ophthalmology’s next-generation business conference that brings together leading ophthalmic companies and important new startups for fresh perspectives. The event was created in partnership between the American Academy of Ophthalmology (AAO) and the American Society of Cataract and Refractive Surgery (ASCRS) and occurs the day prior to the American Academy of Ophthalmology (AAO) 2021 Annual Meeting. For more information, visit www.eyecelerator.com.

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media contact:
Heather Anderson
919-827-5539
handerson@6degreespr.com

 

RD Fund Announces Fundraising Efforts Underway for RD Fund 2

Patient-centric venture philanthropy model continues to invest in companies developing therapies for individuals affected by retinal degenerative diseases.

 

Raleigh, N.C.October 28, 2021 – Today, the RD Fund (Retinal Degeneration Fund) – the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases – announces fundraising efforts are underway for RD Fund 2, targeting at least $75 million in new capital via major philanthropic donations.

RD Fund 2 has the ability to lead investments and the flexibility to make follow-on investments in RD Fund 1 companies. RD Fund 2 will build on the diversity of the overall portfolio, including novel strategies based on modality, disease stage intervention, gene-specific and gene-agnostic approaches to help address as many inherited retinal diseases as possible. The primary focus of the Fund is to invest in therapeutics; however, RD Fund 2 has the ability to make investments in services and technologies that help advance the mission.

“The RD Fund plays an important role in the maturation of the IRD therapeutics field, including academic research projects ready for translation,” said Rusty Kelley, PhD, MBA, senior vice president, investments and alliances of RD Fund and Foundation Fighting Blindness. “Having the ability to conceive new companies, lead their early financing with capital sources from a set of extremely motivated investors, places us in the critical role of de-risking novel technologies that enable clinical and financial returns.” 

To date, RD Fund 2 has raised over $40 million in committed capital, which includes anchor commitments made by Paul Manning and Gordon Gund. Thanks in part to these commitments, Opus Genetics, a recently launched gene therapy-development company conceived internally, raised a $19 million round of seed financing. Opus leverages the expertise of pioneers in ocular gene therapy to advance a wholly-owned portfolio of underserved forms of inherited blindness with an urgent patient-first priority.

"For major contributors in RD Fund 2, there will be the opportunity to be more intimately involved in the funding of highly visible activities in biotech startups and spin-outs who have the goal of accelerating the progress of treatments and cures for inherited retinal diseases," said Gordon Gund, co-founder of the Foundation Fighting Blindness, and chairman and CEO of the Gund Investment Corporation. "It is encouraging to see the RD Fund management team execute on this novel venture philanthropy model. A great example is when RD Fund invested $3 million in Vedere Bio, which was later acquired by Novartis — a committed and skilled large pharmaceutical company in our field — a meaningful return was reinvested in research projects and companies furthering the Foundation's mission."

About the RD Fund

The RD Fund (Retinal Degeneration Fund) is the venture arm of the Foundation Fighting Blindness, and a leading investor in the inherited retinal disease space. It was established in 2018 to serve the Foundations mission to rapidly drive research toward preventions, treatments and cures for the entire spectrum of blinding retinal diseases—including retinitis pigmentosa, macular degeneration, and Usher syndrome. RD Fund focuses on mission-related investments in companies with projects nearing clinical testing. Visit RDFund.org for more information.

 

Media Contact:

Chris Adams

410-423-0585

cadams@fightingblindness.org

Pfizer, Novartis sign on to accelerate gene therapies for rare diseases with US agencies

by Annalee Armstrong | 

Oct 28, 2021 9:55am

 

A cadre of big and small biotechs and pharmas, plus nonprofit organizations, have signed on to a new U.S. initiative to go after gene therapies for rare diseases.

Called the Bespoke Gene Therapy Consortium, or BGTC, the initiative is headed up by the FDA and the National Institutes of Health (NIH) and is designed to accelerate development of gene therapies. The agencies are putting up $76 million over five years.

On the list of companies are Biogen, Johnson & Johnson’s Janssen pharmaceutical unit, Pfizer, the Novartis Institutes for BioMedical Research, Regenxbio, Spark Therapeutics, Takeda, Taysha Gene Therapies, Thermo Fisher Scientific and Ultragenyx. The Alliance for Regenerative Medicine, the American Society of Gene and Cell Therapy and other groups are also involved.

While the diseases are rare, the collective group could make an impact on conditions that affect 30 million Americans. Just two rare diseases out of the 7,000 recognized have approved gene therapies: Novartis’ Zolgensma in spinal muscular atrophy and Spark’s Luxturna for RPE65 mutation-associated retinal dystrophy.

The BGTC, which is part of the NIH Accelerating Medicines Partnership program, will optimize and streamline the development process for gene therapies for rare diseases to help fill the unmet need.

“Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” said NIH Director Francis Collins, M.D., Ph.D. “There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies.”

Developing gene therapies is a highly complex process that takes significant time and resources. Companies tend to tackle one disease at a time, and the right tools can be hard to come by. There’s also no standardization across the field, the FDA release said.

“Rare diseases affect 25 to 30 million Americans, but because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualized gene therapy treatments for a single disease to market,” said Joni Rutter, Ph.D., acting director of NIH’s National Center for Advancing Translational Sciences.

If the companies can come together and standardize a therapeutic development model with a common gene therapy delivery technique—also known as a vector—the process could be more efficient. A main goal of the BGTC will be to better understand the basic biology of the adeno-associated virus vector that delivers gene therapies and develop a standard test to aid manufacturing and enhance therapeutic benefits.

The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivize more companies to invest in this space and bring treatments to patients,” Rutter added.

The initiative will support four to six clinical trials focused on rare, single-gene diseases or commercial programs that are further along in development that could be scaled up quickly. Streamlining the regulatory process will also be part of the BGTC.

There’s plenty of opportunity for companies that do get a rare disease gene therapy across the finish line. Novartis just reported $375 million in sales for Zolgensma for the third quarter amid steady sales in the U.S. and as the therapy became more available across Europe.

Biogen, Novartis and many of the companies on the list all have pipelines full of gene therapies that could meet the rare disease criteria. But the path has been bumpy, with many failures and safety concerns sidelining promising medicines. An FDA advisory committee recently reviewed safety of these therapies and questioned the best animal models to use in early research to ensure human safety once testing advances. 

Opus Genetics Announces Two Key Leadership Appointments

Wed, 20 October 2021, 7:30 am
  • Dr. Ash Jayagopal named Chief Scientific Officer

  • Mr. Joe Schachle appointed Chief Operating Officer

RALEIGH, N.C., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for orphan inherited retinal diseases, today announced two key appointments to its founding executive team. Ash Jayagopal, Ph.D., has joined the company as Chief Scientific Officer (CSO) and Joe Schachle has joined as Chief Operating Officer (COO).

“We were thrilled to launch Opus last month to advance an AAV-based gene therapy portfolio to treat neglected, orphan inherited retinal diseases, and we welcome Ash and Joe’s leadership at this important and foundational time for the Company,” said Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus. “Ash’s career discovering and developing therapies for ocular diseases makes him well-suited for the role of Opus CSO, where he will be instrumental in the advancement of our initial programs OPGx-001 and OPGx-002 for Leber congenital amaurosis. Additionally, Joe brings deep operational experience to his role as COO, specifically his expertise in strategic planning, business development and operations, which will be invaluable as we build our company and create novel orphan manufacturing scale and efficiencies.”

Dr. Jayagopal has more than 13 years of experience in drug development, drug delivery platforms and biomarker development for retinal diseases. Prior to joining Opus, Dr. Jayagopal served as the executive director of discovery medicine at Kodiak Sciences, where he led the drug discovery team and shaped the strategy for leveraging Kodiak’s biopolymer technology for delivery of large and small molecules in retinal diseases. Previously, Dr. Jayagopal was head of molecular pharmacology and biomarkers in ophthalmology at Roche, where he built and led a team of more than 25 scientists focused on the discovery and validation of biologics, small molecules, and gene therapies for ocular diseases, including inherited retinal diseases. Dr. Jayagopal has also served as an assistant professor in the departments of ophthalmology and visual sciences at the Vanderbilt Eye Institute, and the departments of molecular physiology and biophysics of Vanderbilt University Medical Center.

“Opus was formed to bring an unprecedented combination of resources, elite science and the expertise of pioneers in ocular gene therapy to bear and drive transformative treatments to patients,” said Dr. Jayagopal. “With its advanced pipeline – unique for a company at this stage – Opus has the potential to be clinical-stage in the near-term, and I look forward to working with the team to break new scientific ground in the pursuit of better treatments for inherited retinal diseases.”

Mr. Schachle brings more than 30 years of experience in life sciences to Opus, with specific expertise in strategic and operational planning, business development, marketing and sales, and business intelligence. Before joining Opus, Mr. Schachle served as vice president of Customer Experience Enablement and vice president of global commercial services and controlling at Grifols, where he led multiple commercial departments across business units, managed key cross-divisional initiatives and directed strategic brand planning process. Previously, Mr. Schachle was the COO for Parion Sciences, and part of the team that secured partners for the company’s lead programs, which exceeded $1 billion in deal value. Mr. Schachle has also served as chief commercial officer for Inspire Pharmaceuticals, where he oversaw multiple partnering deals and promoted three eye care brands, including Restasis®. In addition, he’s held multiple sales and marketing leadership positions at GlaxoSmithKline, where he managed several billion-dollar brands including Advair®, Imitrex®, Wellbutrin SR®, Epivir® / Retrovir® and Combivir®.

“Opus is built by and for patients to efficiently move validated science toward patients leveraging scalable, strategic manufacturing and processes,” said Mr. Schachle. “I look forward to leading the work to operationalize this first-of-its-kind model to address significant unmet need in the treatment of neglected, orphan inherited retinal diseases.”

Dr. Jayagopal holds a Ph.D. in biomedical engineering from Vanderbilt University and an MBA from the Kelley School of Business, Indiana University. Mr. Schachle holds a B.A. in business administration from James Madison University and an MBA from Old Dominion University.

About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.

Media contact:
Heather Anderson
919-827-5539
handerson@6degreespr.com

Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology

Cambridge, MA, and Paris, France– October 13, 2021 – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, today announced a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases.

As part of this collaboration, Intellia will grant SparingVision exclusive rights to Intellia’s proprietary in vivo CRISPR/Cas9-based genome editing technology for up to three ocular targets addressing diseases with significant unmet medical need. SparingVision will lead and fund the preclinical and clinical development for the genome editing product candidates pursued under the collaboration. In addition, the parties will research and develop novel self-inactivating AAV vectors and LNP-based approaches to address delivery of CRISPR/Cas9 genome editing reagents to the retina.

As part of the licensing agreement, Intellia will receive a 10% equity ownership stake in SparingVision. Intellia will also be eligible to receive certain development and commercial milestone payments (around $200 million per product) as well as royalties on potential future sales of products arising from the collaboration. In addition, Intellia may exercise an option to obtain the US commercialization rights for product candidates arising from two of three collaboration targets. For product candidates Intellia chooses to option, Intellia will pay an opt-in fee, reimburse certain costs, share in 50% of development costs and pay royalties to SparingVision on US sales. Intellia will also maintain the ability to leverage technology advances established under this collaboration for any targets outside the partnership.

Stéphane Boissel, President and Chief Executive Officer of SparingVision, said:SparingVision’s aim has always been to disrupt the ophthalmology field by using cutting-edge technologies to address areas of significant unmet need. This collaboration with Intellia marks a pivotal moment in this mission and is highly complementary to our already mature and growing pipeline of unique mutation-agnostic gene therapies. Intellia is the first company in history to present clinical data supporting precision editing of a disease-causing gene within the body following a single, systemic dose of CRISPR/Cas9 and we are honored to have been selected as a strategic partner. We look forward to working together with the shared goal of radically changing the treatment of blinding ocular diseases.”

Intellia has been a pioneer in utilizing CRISPR/Cas9 technology to develop potentially curative treatments for genetic diseases. Today’s announcement is another step forward in more fully leveraging the power of our genome editing technology to address diseases inadequately treated with existing medicines,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We have been thoroughly impressed with the team at SparingVision, particularly regarding their unparalleled understanding of retinal diseases and trackrecord for developing novel therapies for patients with ocular diseases. We believe SparingVision will be an excellent partner to expand our genome editing capabilities into the field of ophthalmology and we look forward to our new partnership.

The transaction is expected to close in the fourth quarter and is subject to certain closing conditions.

 

About Intellia Therapeutics

Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of diseasecausing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.

 

About SparingVision:

SparingVision is a genomic medicines company, translating pioneering science into vision-saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

 

Intellia’s Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding Intellia’s beliefs and expectations regarding: its strategy, business plans and focus; its ability to quickly and efficiently realize the scope and potential of its genome-editing technology; its ability to maintain, expand and maximize its intellectual property portfolio and pipeline as well as accelerate clinical validation for its platform; the therapeutic value and development potential of CRISPR/Cas9 gene editing technologies and therapies; its ability to combine its CRISPR genome editing platform with SparingVision’s expertise in ocular diseases to create successful therapeutic products; and the expected strategic benefits of any current or future collaborations.

Any forward-looking statements in this press release are based on management's current expectations and beliefs of future events, and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks related to Intellia’s ability to protect and maintain its intellectual property portfolio; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of Intellia’s licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for the new company’s product candidates; the risk that any one or more of the collaboration product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaboration with SparingVision or its other collaborations will not continue or will not be successful. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q filed with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release represent Intellia’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Intellia explicitly disclaims any obligation to update any forward-looking statements, except as required by law.

Intellia beefs up ocular pipeline with 10% stake in SparingVision

by Kyle LaHucik | 

Oct 13, 2021 7:00am

 

Intellia Therapeutics was the first to prove gene editing can work in a human and now the famed CRISPR/Cas9 biotech is beefing up its ocular disease pipeline with a 10% equity stake in SparingVision. 

The first-in-human results, issued in June, made Intellia the "best possible partner," said SparingVision President and CEO Stéphane Boissel in an interview with Fierce Biotech. 

Intellia will provide exclusive rights to its in vivo CRISPR/Cas-9-based gene editing technology to SparingVision for up to three ocular targets. Boissel declined to disclose the specific targets, noting there are more than 250 in the eye they could go after. 

“With Intellia, what we’re hoping to do is to go where the whole field should be going in terms of correcting or dealing with the consequences of mutations directly at the level of the chromosome," said Deniz Dalkara, Ph.D., chief scientific officer of SparingVision, in the joint interview. "Inherited retinal degenerations are caused by a multitude of mutations and some of these mutations are of interest to us as priority targets."

Intellia will collect up to $200 million in biobucks per product that arises from the collaboration and also snags the option to obtain U.S. commercialization rights to products from two of the three targets. SparingVision is responsible for preclinical and clinical development for the gene editing treatments involved in the collaboration.

The companies will also research and develop self-inactivating adeno-associated virus-based vectors and lipid nanoparticle-based ways to deliver the gene editing reagents to the retina. 

This isn't Intellia's first foray into the ocular arena. The company expanded a cell therapy collaboration with Novartis to include ex vivo development using ocular stem cells in December 2018. Under that $10 million cash deal, Novartis has the right to develop CRISPR/Cas9-based treatments for targets using ocular stem cells. Details on the advancement of that work have been kept under the hood.

Aside from working with Novartis on that and sickle cell disease, Intellia is also partnered with Regeneron on inherited blood disorders and the rare disease transthyretin amyloidosis. That's the disease that Intellia reported data on earlier this summer.

Back on the road

SparingVision is not wasting any time getting started on its collaboration with Intellia. 

“We want to go fast and we are very soon going to elect the first target so that we start the development in 2022. Beyond that, that will be a little foolish of myself to give you a timeline," Boissel said. 

Jeito Capital and 4BIO-backed SparingVision went with an equity stake rather than upfront cash "given the price tag of this kind of technology," Boissel said. 

With about €60 million ($69 million) infused into the biotech to date, SparingVision is on par to be "back on the road in the next few months to raise cash," Boissel said. The extra money will be critical to the Intellia deal, if all goes well. 

Don't expect SparingVision to link up with any similar partners for a while as the biotech now has its "hands very full for the next few years," the CEO said. 

The rest of SparingVision's mutation-agnostic pipeline includes a vision deterioration treatment that could land in the clinic "as soon as next year" with a GLP tox study beginning next month, Boissel said. Behind that is an asset that could enter human studies in 2024, the CEO added.

 

RD Fund Appoints Tony Adamis, MD to Board of Directors

 

Raleigh, N.C.October 4, 2021 – Today, the Retinal Degeneration Fund (RD Fund) – the venture arm of the Foundation Fighting Blindness and leading investor in the inherited retinal disease space – announced the appointment of Anthony (Tony) Adamis, MD, to its board of directors. Dr. Adamis, who most recently served as SVP of Development Innovation at Genentech, brings over 30 years of industry R&D experience to the role.

“We’re thrilled to welcome Dr. Adamis to the RD Fund and look forward to leveraging his robust industry expertise to further drive research toward preventions, treatments, and cures for the entire spectrum of blinding retinal diseases,” said Ben Yerxa, CEO of RD Fund and the Foundation Fighting Blindness. “As we operate in a rapidly changing environment of technological advancements with many more academic research projects ready for translation, the success of our venture philanthropy model relies heavily on the type of strategic partners we are able to bring to the table. We know Dr. Adamis will help expand RD Funds reach.”

“We strive to recruit a high caliber, engaged board of directors who can bring a diverse set of business, finance, health care, and research expertise to the table,” said Warren Thaler, RD Fund board chairman. “With his deep research and industry experience, Dr. Adamis is a welcome addition in our work to deliver more treatments and cures to the patients who need them.”

Adamis is best known for his co-discovery of the role of vascular endothelial growth factor (VEGF) in ocular disease, including diabetic retinopathy and wet age-related macular degeneration (AMD). This research, conducted at Harvard in the 1990s, led to his sharing the António Champaulimaud Vision Award in 2014. In 2000, he co-founded Eyetech Pharmaceuticals, which developed and obtained FDA approval for the first anti-VEGF drug in ophthalmology (pegaptanib for AMD; 2004).

At Genentech (2009 – present), Adamis helped lead the teams that developed the first FDA-approved drugs for diabetic macular edema, branch and central retinal vein occlusion, diabetic retinopathy, and myopic choroidal neovascularization (ranibizumab, anti-VEGF), as well as temporal arteritis (tolicizumab, anti-IL6R). Since the introduction of anti-VEGF drugs, the rates of blindness from wet AMD have dropped by half across the world.

He received his MD with Honors from the University of Chicago and completed his ophthalmology residency at the University of Michigan and his fellowship at the Massachusetts Eye and Ear Infirmary. Adamisresearch training in vascular biology was with Dr. Judah Folkman at the Boston Childrens Hospital.

“RD Fund is a unique player in the eye disease investor space with its ability to activate the Foundations 50-year research track record to make sound investments in pre-clinical start-ups, which is proving to be a successful model,” said Dr. Adamis. “Im excited to join the RD Funds board and support its efforts to translate more treatments and cures from bench to bedside.”

 

About The Retinal Degeneration Fund

The Retinal Degeneration Fund (RD Fund) is the venture arm of the Foundation Fighting Blindness, and a leading investor in the Inherited Retinal Disease space. It was established in 2018 to serve the Foundations mission to rapidly drive research toward preventions, treatments and cures for the entire spectrum of blinding retinal diseases—including retinitis pigmentosa, macular degeneration, and Usher syndrome. RD Fund focuses on mission-related investments in companies with projects nearing clinical testing. Visit RDFund.org for more information.

 

Media Contact:

Chris Adams 

 

cadams@fightingblindness.org

 

ProQR Appoints Theresa Heggie as Chief Commercial Officer

  • Senior leader with extensive global rare disease commercialization experience
  • Top-line data from Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10 on track for H1 2022

LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 04, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced the appointment of Theresa Heggie as Chief Commercial Officer reporting to Daniel de Boer, Founder and CEO. In this newly created leadership position, Ms. Heggie will be responsible for overseeing the Company’s commercial strategy and global commercial operations. She joins as the Company prepares for the top-line results from its Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10 in the first half of 2022.

“As we prepare for results from our Phase 2/3 trial of sepofarsen, we are delighted to have Theresa join ProQR as the head of our commercial organization,” said Daniel A. de Boer, Founder and CEO. “Theresa brings a wealth of commercial and leadership skills to ProQR, having built commercial organizations and successfully launched orphan products with Alnylam and Shire. Theresa has served on our Supervisory Board for the last few years and is uniquely qualified to help us as we work to advance our pipeline of therapies for patients with genetic eye disease.”

“I am delighted to be joining ProQR at such an exciting time for the Company,” said Ms. Heggie. “Having served on the Supervisory Board since 2019, I have seen first-hand the commitment ProQR has to provide safe and effective treatments to patients with genetic eye diseases. I look forward to partnering with Daniel and the ProQR team to prepare for commercialization and to working with the team, employees, physicians, and patient organizations to bring these therapies to patients.”

With significant experience in the global commercialization of rare diseases, as well as in RNA therapeutics, Ms. Heggie most recently served as Chief Executive Officer of Freeline Therapeutics. She previously served in senior commercial and operating roles at both Alnylam Pharmaceuticals as Senior Vice President, Head of CEMEA, and at Shire where she built the EMEA rare disease business and subsequently led the Global Commercial Operations for rare diseases. Following Shire’s acquisition of Jerini, she served as its Chief Executive Officer. Ms. Heggie currently serves on the board of BioCryst Pharmaceuticals. She received a BSc from Cornell University.

In connection with her appointment as Chief Commercial Officer, Ms. Heggie has stepped down from the Supervisory Board of ProQR.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of genetic eye diseases, with a focus on inherited retinal diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com.

Forward Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding sepofarsen (QR-110) and the clinical development and the therapeutic potential thereof, including timing of clinical trials and results from such clinical trials. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; and general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

For ProQR Therapeutics N.V.

Investor Contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Hans Vitzthum
LifeSci Advisors
T: +1 617 430 7578
hans@lifesciadvisors.com

Media Contact:
Cherilyn Cecchini, MD
LifeSci Communications
T: +1 646 876 5196
ccecchini@lifesciadvisors.com

RD Fund Launches Opus Genetics with $19M Seed Funding to Advance Gene Therapy Treatments for Blinding Conditions

  • Initial programs will focus on treatments for rare pediatric blinding conditions
  • Company formed to advance the work of scientific cofounders Dr. Jean Bennett, Junwei Sun and Dr. Eric Pierce

Raleigh, N.C. – September 22, 2021 – The Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases, today announced the launch of Opus Genetics, a patient-focused gene therapy company efficiently developing therapies for orphan inherited retinal diseases. The $19 million in seed financing was led by the RD Fund with participation from the Manning Family Foundation and Bios Partners. 

This is the first spin-out company internally conceived and launched by the RD Fund to further the Foundation’s mission. The initial seed funding will allow Opus to advance the preclinical research of its scientific founders, Jean Bennett, M.D., Ph.D., the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania, and Junwei Sun, chief administrator of Penn’s Center for Advanced Retinal Ocular Therapeutics (CAROT), and Eric Pierce, M.D., Ph.D., William F. Chatlos Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear. Dr. Bennett and Mr. Sun are also members of the Spark Therapeutics founding team.

“I’ve dedicated my career to the research and development of treatments for blinding diseases, and I’m eager to continue to build on this work with the RD Fund, an organization that understands the science and is deeply ingrained in the patient community,” said Dr. Bennett. “Founding Opus enables us to progress our first two programs in Leber congenital amaurosis while building an engine to move additional treatments toward the patients who need them.”

The company’s lead programs are licensed from the University of Pennsylvania and will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). LCA is a group of rare inherited retinal diseases that typically present in infancy and are characterized by degeneration of photoreceptors, the cells in the retina that make vision possible. Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. LCA5 is one of the most severe forms of LCA, and affects approximately one in 1.7 million people. The company’s second program, OPGx-002, will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13), which affects one in 288,000 people. Recent preclinical data have demonstrated the potential for both of these novel approaches to restore structure and function. Opus expects to file an IND for its OPGx-001 program in early 2022, and enter the clinic in mid-2022.

 “Opus is a first-of-its-kind model for patient-focused therapeutic development. As the first company launched by the Foundation’s venture arm RD Fund, Opus is uniquely positioned to bring experts, resources and patients together to efficiently advance ocular gene therapies for small groups of patients that to date have been neglected,” said Ben Yerxa, Ph.D., CEO of the Foundation and the RD Fund, and acting CEO of Opus. “We’re grateful for our fellow investors and supporters who share our commitment to realizing the promise of improving vision for people with devastating sight-limiting diseases, and look forward to building upon the pioneering work of Dr. Bennett, Mr. Sun and Dr. Pierce, and expanding our pipeline with more programs soon.”

In addition to Dr. Yerxa, the company is co-founded and managed by Rusty Kelley, Ph.D., Peter Ginsberg, and Jason Menzo, who also form the management team of the RD Fund. The Board for Opus is comprised of Drs. Yerxa, Kelley and Bennett.   

“While potential treatments for these ultra-rare conditions have existed for years, families have been stuck in a holding pattern waiting on someone to deliver a feasible business model to bring them to market,” said Paul Manning, Manning Family Foundation. “We’re thrilled to be a part of the launch of Opus Genetics to establish a patient-first priority and build capabilities to tackle manufacturing obstacles and access to life-altering treatments for the people who need them most.”

For more information, visit www.opusgenetics.com.  

About Opus Genetics

Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Raleigh, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com

About The Retinal Degeneration Fund

The Retinal Degeneration Fund (RD Fund) is the venture arm of the Foundation Fighting Blindness, and a leading investor in the Inherited Retinal Disease space.   It was established in 2018 to serve the Foundation’s mission to rapidly drive research toward preventions, treatments and cures for the entire spectrum of blinding retinal diseases—including retinitis pigmentosa, macular degeneration, and Usher syndrome. RD Fund focuses on mission-related investments in companies with projects nearing clinical testing.

About Foundation Fighting Blindness

Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $750 million toward its mission of preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit www.FightingBlindness.org for more information.  

 University of Pennsylvania Financial Disclosure

Jean Bennett and Junwei Sun are scientific collaborators, advisors and co-founders of Opus Genetics. As such, they hold an equity stake in the Company, and as inventors of the licensed technology may receive additional future financial benefits under licenses granted by Penn to Opus Genetics. Dr. Bennett’s laboratory at Penn receives sponsored research funding from Opus Genetics. The University of Pennsylvania also holds equity and licensing interests in Opus Genetics.

Media contacts:

For The Retinal Degeneration Fund:

Jason Menzo

317-489-7283

jmenzo@FightingBlindness.org

For Opus Genetics:

Heather Anderson

919-827-5539

handerson@6degreespr.com

Luxturna inventor Jean Bennett starts a new gene therapy company to tackle rare diseases left behind by pharma, VCs

Endpoints

By Jason Mast

Sept. 22, 2021

 

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

The problem, they explained, was simple: The diseases she was trying to treat were devastating but too rare to be worth the cost of investment.

“There’s lots of interest in diseases like glaucoma and age-related macular degeneration, where millions of people are affected,” Bennett said. “But when you’re talking about diseases where there may only be [2,000] people affected in the US — that’s hard.”

So Bennett, after a series of connections, began building her own company, with people whose job it was to care about these ultra-rare eye diseases and who had money to do so. On Thursday, she and the Retinal Degeneration Fund, the venture arm of the Foundation for Fighting Blindness, unveiled Opus Genetics.

Seeded with $19 million, mostly from RD Fund, the company will try to bring two of Bennett’s therapies for rare forms of blindness into the clinic in the next two years, while licensing in other programs for rare vision diseases that academics can’t get funding for or pharmas have deprioritized.

“There were gene therapy assets out there that weren’t getting the attention they needed and needed to find a home,” said Ben Yerxa, CEO of the foundation and interim CEO of Opus. “These weren’t just orphan assets, they were orphaned assets.”

Opus is part of a series of for-profit and not-for-profit efforts — Opus decidedly on the for-profit side — that have popped up to bring gene therapy to patients who seem left out of the industry for the field’s future. Although rare single-gene conditions like leber congenital amaurosis, the condition Luxturna treats, provided the proof-of-concept for gene therapy, investors and large companies are increasingly focused on applying the tech to larger (and more lucrative) diseases.

That push could leave behind patients with diseases that would otherwise be a perfect fit for gene therapy. One of the diseases Opus is pursuing is a subset of Leber that actually affects more patients than the Leber subset that Luxturna treats. But even Spark Therapeutics, the company Bennett co-founded to take Luxturna to market, has moved on to more common disorders such as hemophilia and Huntington’s.

“It’s not as interested in ultra-rare conditions, as far as I know,” Bennett said.

In extreme cases, a program can be abandoned even after companies have spent millions developing it, in part because just manufacturing and administering gene therapy is costly. Earlier this year, Orchard Therapeutics published data showing its gene therapy for severe combined immune deficiency was nearly 100% effective. But the biotech had already decided to shuttle the program for a condition that affects only between 1 in every 200,000 to 1 in every 1 million births.

Jim Wilson and Tachi Yamada set up an attempt at a non-profit solution this year that will try to exploit the market value of priority review vouchers the FDA gives out to make gene therapy sustainable for obscure conditions. In 2017, Wilson’s former protege, Luk Vandenberghe, set up his own non-profit to get rare disease gene therapies to a stage companies can take them on.

Opus, though, will try to make developing therapies for these rare vision disorders profitable by building a portfolio of them, de-risking and lowering the cost of each. It’s a similar approach to the one that turned Taysha into a nearly billion-dollar company overnight, Yerxa said, but for the eye instead of the central nervous system.

Opus also currently has 24 fewer programs than Taysha, but Yerxa said that will eventually change.

“We pretty much fund the space,” he said, referring to the foundation’s longstanding work advancing basic research on vision disorders. “We fund like 90 labs and we’ve got a pretty good handle on which labs are producing great programs.”

For now, the company is a tiny operation; Yerxa and three other members of the foundation are staffing it on top of their day jobs. But he said they will announce two permanent C-suite members shortly. The seed funding will last 18-24 months.

Although gene therapy has seen its share of safety concerns over the past year, Opus should see smoother sailing. Because the eye is a largely self-contained unit, it is easier and safer to deliver there.

The company’s first program, for LCA5 mutations of Leber, is scheduled for the clinic at the beginning of next year. The second, for LCA13 mutations, is about a year behind.

The former affects around 1 in 1.7 million people, the latter around 1 in 288,000 — both highly rare but far from unique. Plenty of other diseases, affecting other organs, are similarly uncommon and could also use a path to the market.

“We think we have a path forward,” Bennett said. “We want to develop a model.”

To Get New Treatments Across the Finish Line, a Funder Fighting Blindness Turns to Investing 

July 29th, 2021 - Source: Inside Philanthropy

 

Since its establishment in 1971 by a group of families affected by retinal disease, the Foundation Fighting Blindness has funded research into several of the most serious and common of these conditions, like macular degeneration and retinitis pigmentosa. The foundation grew to become the leading private funder of basic research into conditions that cause blindness and vision loss for more than 10 million Americans and millions more worldwide. In its 50 years in operation, the foundation has raised over $816 million to support research—including the identification of key genes linked to retinal diseases—that has enabled the launch of dozens of clinical trials to test possible treatments. All in all, a solid track record.

Click here for the rest of the article

Atsena Therapeutics Appoints Jennifer Wellman to Board of Directors

 

DURHAM, N.C., July 12, 2021 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the appointment of Jennifer Wellman to its board of directors. Ms. Wellman, a biotech executive with more than 20 years of gene therapy development experience, will serve as an independent director.

“Jen’s extensive U.S. and EU regulatory expertise and impressive track record in the clinical development of AAV-based gene therapies are valuable to Atsena as we progress cutting-edge gene therapies designed to overcome the hurdles presented by inherited retinal disease,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “We’re thrilled to welcome Jen to Atsena’s board of directors and look forward to leveraging her strategic insights.”

Ms. Wellman is the Chief Operating Officer of Akouos, Inc., a precision genetic medicine company. Prior to Akouos, Ms. Wellman was Head of Product Development Strategy at Spark Therapeutics, Inc., now a subsidiary of Roche Holding AG, where she was also a co-founder. At Spark, she led the regulatory and clinical development for LUXTURNA® from the pre-IND phase through the filings of the marketing applications to the U.S. Food and Drug Administration and European Medicines Agency. Previously, Ms. Wellman was the Director of Regulatory Affairs for the Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia; there, she directed multiple AAV preclinical programs and clinical trials for inherited retinal diseases, as well as other therapeutic areas. Earlier in her career, she served as an Associate Scientist at Avigen, Inc. Ms. Wellman received her M.S. from the University of New Haven and B.S. in microbiology and immunology from Queens University (Canada).

“Atsena’s clinical program for LCA1 and novel AAV capsid technology position the company to deliver potential life-changing treatments uniquely suited to prevent or reverse blindness and vision loss,” said Ms. Wellman. “I’m delighted to join Atsena’s board and support the team in the development of therapies for individuals living with inherited retinal diseases.”

Benjamin Yerxa, PhD, Chief Executive Officer of Foundation Fighting Blindness, has moved from Atsena’s board of directors to its scientific advisory board.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating a potential therapy for one of the most common causes of blindness and vision loss in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated viral (AAV) vector technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
tplohoros@6degreespr.com

Business Contact:
info@atsenatx.com

Venture philanthropy: The secret weapon for unlocking biomedical research's full life-changing potential

June 04, 2021

First published in Philanthropy News Digest's PhilanTopic blog. Click here for the original article.  

Eye_retina_gettyimages_batke

More than a year into the COVID-19 pandemic, there has been much reflection around "lessons learned" across all sectors. In the biomedical research space, we've seen science meet the urgent need for safe and effective vaccines at miraculous speed to contain the spread of the virus. The mRNA technology used in some of those vaccines has broad implications for future treatments for a variety of other viruses, cancers, and diseases and is a clear indication of how far science has evolved in a short period of time. Imagine what treatments and cures could be unlocked — with the necessary funding.

In the United States, public funding for basic research has long come from the National Institutes of Health, but the U.S. government lags other advanced economies in the amount of funding it provides for the translational research required to convert basic science into tangible patient treatments. And while more public funding for biomedical research at the critical clinical trial stage is essential, it is going to take public, private, and philanthropic dollars to ensure that biomedical research into promising treatments and cures doesn't wither on the vine. Federal programs such as the Cancer Moonshot, state-level initiatives like the California Institute for Regenerative Medicine, and promising legislation aimed at providing private-sector loans to companies developing novel treatments for disease and disability are all helpful — but still leave a funding gap. There needs to be a third leg to stabilize those public- and private-sector efforts, and we believe that third leg is philanthropy.

As successful entrepreneurs and venture investors, we see our donations as investments in the mission of the nonprofit organizations we support. We each have a personal connection to the mission of the Foundation Fighting Blindness: one of us has experienced loss of sight from retinitis pigmentosa as a young adult, and the other has raised two sons with vision impairment caused by Stargardt disease. Based on our personal experiences, we have a keen understanding of what it is like to be a patient or have a loved one waiting for life-changing treatments to become available.

For fifty years, thanks to the generosity of donors, the Foundation Fighting Blindness has successfully funded research in pursuit of treatments and cures for the entire spectrum of inherited retinal diseases (IRDs) and dry age-related macular degeneration (AMD), which together affect more than two hundred million people globally. Yet, more needs to be done. The key discoveries made in labs need to make it into the hands of industry-led therapy developers to conduct clinical testing and win FDA approval. But a gap in funding often prevents this progress, and in this case, the science is now outpacing the funding.

To bridge this funding gap, the Foundation Fighting Blindness created the Retinal Degeneration Fund (RD Fund), a nonprofit, pure-play venture philanthropy investment vehicle designed to help accelerate the technical aspects of the organization's mission and advance its financial goals. Our respective family foundations contributed significant capital to launch the fund, which allowed us to be more involved in the organization's work by funding highly visible activities in biotech startups and spinouts. We've taken concepts and techniques from our venture capital finance and business management experience and applied them to our philanthropic goals of accelerating the progress on treatments and cures, while positioning the organization for long-term sustainability.

Launched in late 2018 with $72 million under management, the first fund is now 90 percent committed, with nine investments plus reserves. This invested capital has attracted an additional $400 million in capital to date from institutional co-investors and has produced its first exit with the sale of Vedere Bio to Novartis for $280 million, enabling the organization to plug a financial gap in its long-range science spending plan and roll over significant funds to seed Fund 2. 

We take comfort in knowing that the venture philanthropy model already has been successfully scaled by the Bill & Melinda Gates Foundation, the Cystic Fibrosis Foundation, and the Juvenile Diabetes Research Foundation, just to name a few. One key element is to manage it professionally and deliberately; one cannot just wander into biotech equity investing without experience, deep scientific know-how, and world-class advice and oversight. The RD Fund has an independent board of directors with expertise spanning retinal biology, clinical ophthalmology, finance, and entrepreneurship, and the board works closely with an executive management team with significant operational, strategic, and leadership experience. Importantly, the fund is able to rely on an international scientific advisory board and leverage the organization's patient registry and clinical consortium. In other words, the brain trust of the Foundation Fighting Blindness and its venture arm have the collective scientific and business acumen to best determine what is or is not an investible mission-related opportunity.

We are encouraged by venture philanthropy's ability to reap a return to be re-invested in furthering an organization's mission, especially in times of economic uncertainty. Most important, our experience has demonstrated that jump-starting the pipeline for treatments and cures through venture philanthropy holds real promise as a viable, scalable approach for addressing other underserved diseases impacting so many.

(Photo credit: GettyImages/Batke)

Gordon Gund is chair and CEO of Gund Investment Corporation; after losing his sight from retinitis pigmentosa in 1970, he co-founded the Foundation Fighting Blindness with his wife, Lulie, and others. Paul Manning is founder, chair, and CEO of PBM Capital; both of his sons were diagnosed with Stargardt disease.

Gordon Gund_Paul_Manning_PhilanTopic

Vedere Bio II Launches with $77 Million Series A Financing to Develop Next Generation Ocular Gene Therapies

Uses novel, mutation agnostic optogenetics approach to restore vision in underserved forms of blindness

Company backed by leading life science investors including Octagon Capital, Samsara BioCapital and Casdin Capital, along with founding investors Atlas Venture, Mission BioCapital and Foundation Fighting Blindness


CAMBRIDGE, Mass., May 18, 2021 – Vedere Bio II, Inc., a company developing next generation ocular gene therapies designed for vision restoration and preservation for patients with vision loss due to photoreceptor death, today announced the completion of its $77 million Series A financing. Founded by the leadership and research team behind the former Vedere Bio, which was acquired by Novartis in September 2020, Vedere Bio II (Vedere) will leverage its new, proprietary, mutation agnostic optogenetics technology to improve upon current gene therapies by restoring functional vision to patients. The financing was led by Octagon Capital, who was also joined by new investors Samsara BioCapital and Casdin Capital, and Vedere’s founding investors, Atlas Venture, Mission BioCapital and the RD Fund, the venture arm of Foundation Fighting Blindness.

“The launch of Vedere Bio II represents a milestone moment in our work to restore vision to patients with both genetic and non-genetic causes of vision loss, and we are excited to work with both our new and founding investors to advance our pipeline,” said Cyrus Mozayeni, M.D., Chief Executive Officer, President of Vedere Bio II and Atlas Venture Entrepreneur in Residence. “Our novel vision restoration approach targets underserved indications and holds great promise to restore lost vision, exceeding the limitations of traditional gene therapy which primarily aim to slow further vision loss.” 

Vedere aims to increase the quality of vision restoration and preservation for all patients with vision loss due to photoreceptor death through delivery of novel payloads via proprietary intravitreally delivered AAV capsids. While most current gene therapies are targeted to specific gene mutations and only slow down vision loss, Vedere’s technology is mutation agnostic and has the potential to rapidly add new function regardless of disease stage. Vedere’s novel approach is anticipated to have several distinct advantages over other optogenetics approaches, which could help patients see stationary and moving objects in both bright and dim settings without the need for a vision-enhancing medical device. The Series A financing will enable Vedere to advance its platform and therapeutic programs (including the lead program to IND filing), attract talent and invest in research.

“Vedere has a clear mission to restore and preserve vision in patients, and we are excited by the opportunity to partner with and support such a dedicated team with a proven track record of success,” said Ting Jia, Founder and Chief Investment Officer at Octagon Capital. “The company’s unique approach and groundbreaking technology has the potential to address challenges with existing gene therapies that have been unable to be overcome in the past, with the ultimate goal of restoring vision for patients who have limited treatment options.”

Based on technology from the laboratories of Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, and technology directed at enhanced ocular gene therapy delivery arising jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania, Vedere Bio II has developed an optogenetics-based platform to develop treatments for inherited retinal diseases (IRDs) and geographic atrophy (GA), unbound by the specific underlying genetic cause. The therapies confer light sensing properties to cells downstream of photoreceptors, which are preserved in most IRDs and GA, therefore rapidly adding new function, regardless of disease stage.

“Vedere’s next generation optogenetic approach aims to make vision restoration and preservation a reality in largely underserved indications,” said Ben Yerxa, Ph.D., CEO of Foundation Fighting Blindness and the RD Fund. “Inherited retinal diseases and geographic atrophy affect over seven million people globally, and all of these patients deserve life-changing therapies. Vedere’s cutting-edge technology has the potential to dramatically expand the number of patients who can be treated for vision loss caused by photoreceptor cell death.”

About Vedere Bio II, Inc.

Vedere Bio II is a privately held, emerging biopharmaceutical company leveraging mutation agnostic technology and novel AAV capsids to restore vision in all patients with vision loss due to photoreceptor cell death. Comprised of a diverse team of pioneering scientists, Vedere Bio II is discovering and developing next generation ocular gene therapies to increase the quality of vision restoration and preservation for large, underserved indications. The company is headquartered at LabCentral in Cambridge, MA and is funded by Atlas Venture, Casdin Capital, Mission BioCapital, Octagon Capital, the RD Fund and Samsara BioCapital. For more information, please visit www.vederebio.com or follow Vedere Bio II on Twitter and LinkedIn.

  • QR-421a demonstrated a concordant benefit in multiple measures of vision, including best corrected visual activity (BCVA), static perimetry, and retinal imaging (OCT)
  • QR-421a observed to be well tolerated with no serious adverse events reported
  • Two pivotal Phase 2/3 trials are expected to start by the end of 2021
  • Management to host a conference call today at 8:15am EDT

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 24, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. In the trial, QR-421a demonstrated benefit on multiple measures of vision that moved in concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a single dose. QR-421a was observed to be well tolerated with no serious adverse events reported. Based on these findings, the Company plans to advance QR-421a to two parallel pivotal Phase 2/3 trials by year end 2021 – one in early-moderate patients, one in advanced patients.

“We’re pleased to have met all the objectives we set for the Stellar trial, including determining suitable registration endpoints, the dose, dosing interval, and patient population for the Phase 2/3 pivotal trials,” said Aniz Girach, MD, Chief Medical Officer of ProQR, “With just a single dose, QR-421a demonstrated clinical proof of concept with benefit observed in treated eyes compared to the untreated eyes in multiple concordant measures of vision. As expected, we saw benefits in both advanced and early-moderate patients in this slow progressing, debilitating eye disease, allowing us to advance this important investigational therapy for all patients with Usher syndrome and nsRP due to USH2A exon 13 mutations. Based on preliminary Regulatory guidance, we plan to submit protocols to advance QR-421a to pivotal testing. This is our second program targeting a severe inherited retinal disease that is moving into pivotal trials, which we believe further validates our RNA therapy platform and our capabilities to design and efficiently take these programs through clinical development.”

“The safety profile and efficacy findings for QR-421a are very encouraging,” said Robert Koenekoop, MD, MSc, PhD, FRCS(C), FARVO, a clinical-scientist from the Montreal Children’s Hospital and Professor of the McGill University Faculty of Medicine and Department of Pediatric Surgery. “Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A exon 13 mutations are devastating retinal diseases representing a high unmet medical need, as there are no approved therapies to treat the severe vision loss associated with these diseases. Patients’ biggest hope for a therapy is to stop disease progression and prevent vision loss, and these findings suggest that QR-421a has the potential to stabilize vision. I look forward to this exciting program advancing into pivotal trial development.” 

Results from the Phase 1/2 trial of QR-421a

Safety Data

QR-421a was observed to be well tolerated at all doses. There were no serious adverse events reported and no inflammation was observed. One patient had worsening of pre-existing cataracts in both the treated and untreated eyes; both were deemed not treatment related by the investigator. One patient had progression of pre-existing cystoid macular edema (CME) that was managed with standard of care. Both cataracts and CME are associated with a high rate of occurrence in the natural history of this disease.

Efficacy Data

Given the key differences in baseline characteristics, patients were categorized into “advanced” and “early-moderate” populations based on baseline visual acuity.

In advanced patients, the primary measure of efficacy is BCVA. In early-moderate patients, the primary measure of efficacy is measurement of visual fields by static perimetry. QR-421a-treated patients responded on endpoints consistent with their disease stage in both advanced and early-moderate patient populations after a single injection.

All three doses studied in the Stellar trial were observed to be active as predicted by the pre-clinical data. No differences were observed based on patients being homozygous or heterozygous, or having Usher syndrome or non-syndromic retinitis pigmentosa. These findings are consistent with the preclinical data for QR-421a.

Analysis of advanced patients

 

Visual acuity

Best corrected visual acuity, or BCVA, is a measure of central vision, or sharpness of sight, as measured on an Early Treatment of Diabetic Retinopathy Study (ETDRS) letter chart.

Across all treated patients (n=14), a mean benefit of 6.0 letters was observed at week 48 in the treated eyes compared to the untreated (contralateral) eyes after a single injection.

Among advanced disease patients (n=6), a mean benefit of 9.3 letters was observed at week 48 in the treated eyes as compared to the untreated eyes and the benefit was maintained for >12 months. All six advanced patients had a benefit in the treatment eye, whereas none of the patients in the sham group had a benefit in the treatment eye.

Analysis of early-moderate patients

 

Static perimetry

Static perimetry assesses visual fields and retinal sensitivity in the peripheral retina. 

Across all treated patients, the mean total retinal sensitivity improvement was up to 40dB higher in the treated eyes compared to the untreated eyes, and the benefit was maintained for >6 months after a single injection.

The mean number of retinal locations (loci) that improved by ≥7db in retinal sensitivity demonstrated a benefit in the treated eyes compared to the untreated eyes, with up to a mean of 9 loci in the treated eyes improving by ≥7db .

In early-moderate patients (n=8), up to a mean of 13 loci in the treated eyes improved by ≥7db compared to 7 loci for the untreated eyes at the same timepoint.

Concordant benefits were noted on OCT-based assessment of the Ellipsoid Zone layer, which is an objective evaluation of photoreceptor viability, and other measures of central visual function, such as microperimetry.  Sham treated eyes responded similarly to the untreated eyes across all endpoints.

Pivotal trials

On the basis of these findings, the Company plans to conduct two pivotal Phase 2/3 clinical trials. Based on initial Regulatory guidance, the Company plans to submit protocols to start two Phase 2/3 trials. Each trial could potentially serve as the sole registration trial depending on the findings. Pending finalization of the study designs with Regulatory authorities, the trials are expected to start before year end 2021. Both trials are expected to be conducted at global centers of excellence.

Sirius trial in advanced population


The "Sirius" trial is a Phase 2/3 study that will focus on advanced patients with baseline BCVA ≤20/40. The preliminary design for Sirius is a double-masked, randomized, sham-controlled, 24-month, multiple-dose study. The trial is expected to enroll approximately 100 adults with Usher syndrome and nsRP due to USH2A exon 13 mutations, including both homozygous and heterozygous patients. The primary endpoint in this trial will be BCVA at 18 months, with potential for an earlier interim analysis. In this three-arm study, two different doses will be studied that will be administered every 6 months, and a third arm will receive sham treatment.

Celeste trial in early-moderate population

In parallel to Sirius, the Company plans to start the “Celeste” Phase 2/3 trial in early-moderate patients. The preliminary design for Celeste is a double-masked, randomized, sham-controlled, 24-month, multiple-dose study. The trial is expected to enroll approximately 100 adults with Usher syndrome and nsRP due to USH2A exon 13 mutations. The primary endpoint in this trial will be based on static perimetry at 18 months, with potential for an earlier interim analysis. In this three-arm study, two different doses will be studied that will be administered every 6 months, and a third arm will receive sham treatment.

“There are currently no available treatments for the more than 16,000 patients with Usher syndrome 2A and nsRP due to USH2A exon 13 mutations and we are excited about the potential for QR-421a to address this significant unmet need,” said Benjamin R. Yerxa, PhD, Chief Executive Officer at the Foundation Fighting Blindness. “We are pleased to see QR-421a advancing to pivotal testing and proud to support the work of ProQR as they advance their pipeline of RNA therapies to potentially help children, adults, and families who are affected by blindness caused by USH2A mutations and other rare inherited retinal diseases.”

Conference call

Management will discuss the data during a webcasted conference call today at 8:15 am EDT. The dial-in details for the call are +1 631-510-7495 (US), +31 (0)20 714 3545 (NL), conference ID: 8596733.

An archive of the webcast will be available for approximately 30 days following the presentation date.

Phase 1/2 Stellar trial of QR-421a

The Stellar trial is a randomized, sham-controlled, single ascending dose, global, multicenter, 24-month study. The study includes a total of 20 patients, of which 14 received a single dose of QR-421a and six received a single sham procedure for masking. The 14 QR-421a-treated patients enrolled (mean age of 46 years) varied in their disease stage and were classified as advanced patients (defined as patients with baseline visual acuity of <70 letters, or equivalent to LogMAR 0.3, or worse than 20/40 on a Snellen chart) or early-moderate patients. Six patients had advanced disease and eight patients had early-moderate disease. Three different dose levels were studied. The population also varied in disease characteristics with both Usher syndrome (n=7) and nsRP (n=7) and genetic background with both homozygous (n= 9) and heterozygous (n=5) subjects for USH2A exon 13 mutations. The majority of the patients were followed for up to 48 weeks, with one patient followed up to 96 weeks.

About Usher Syndrome Type 2a and Non-Syndromic Retinitis Pigmentosa

Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2a are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a related disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2a and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

About QR-421a

QR-421a is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

Learn more about ProQR at www.proqr.com.

Forward Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof, our other programs and business operations, including timing of commencing clinical trials and enrollment of patients therein, the design of planned trials for QR-421a and the expected regulatory pathway for this product candidate, including the potential for the Sirius and Celeste trials to serve as the sole registration trials in this indication, the expected impact of the COVID-19 on our business operations, including our research and development plans and timelines, and the supply chain for our clinical and development programs. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; and general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Hans Vitzthum
LifeSci Advisors
T: +1 617 535 7743
hans@lifesciadvisors.com

Media Contact:
Cherilyn Cecchini, MD
LifeSci Communications
T: +1 646 876 5196
ccecchini@lifescicomms.com

Paris, March 19, 2021 – SparingVision (“the Company”), a genomic medicine company developing vision saving treatments for ocular diseases, announces today that three abstracts highlighting the company’s recent research into ocular diseases and its lead gene therapy treatment SPVN06 have been accepted for the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, to be held virtually from 1-7 May. The three abstracts will be given as poster presentations for which the details can be found below.

Title: SPVN06, a Novel Mutation-Independent AAV-based Gene Therapy, Protects Cone Degeneration in a Pig Model of Retinitis Pigmentosa
Date and Time: May 3, 2021 from 11:15 AM to 1:00 PM EDT

Presenter: Dr. Jennifer Noel, University of Louisville
Session Title: Drug delivery and Gene Therapy

Title: Correlations between progression markers in rod-cone dystrophy due to mutations in RHO, PDE6A, or PDE6B
Date and Time: May 3, 2021 from 4:30 PM to 6:15 PM EDT

Presenter: Dr. Daniel Chung, Chief Medical Officer, SparingVision
Session Title: Visual Impairment - Assessment and Measurement

Title: A 1-Month Toxicology and Biodistribution NHP Pilot Study Evaluating a Single Subretinal Bilateral Administration of SPVN06 - A Novel AAV-Based Gene Therapy for the Treatment of Rod-Cone Dystrophies Agnostic of the Causative Mutation
Date and Time: May 5, 2021 from 2:45 PM to 4:30 PM EDT

Presenter: Dr. Melanie Marie, SparingVision
Session Title: AMD and retinal physiology

**ENDS**

Contacts:

SparingVision
Stéphane Boissel

Nathalie Trepo 
President and CEO Investor Relations 
nathalie.trepo@sparingvision.com

Consilium Strategic Communications
Amber Fennnell, Olivia Manser, Alexander Bridge
+ 44 (0)20 3709 5700 
sparingvision@consilium-comms.com

 

NOTES TO EDITORS:

About SparingVision:
SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

About SPVN06:
SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVision’s primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2021.

  Former Spark Therapeutics head of ophthalmic medical affairs, responsible for the development and approval of Luxturna™, to lead development of SparingVision’s proprietary asset, SPVN06, and further pipeline development

Paris, February 04, 2021 – SparingVision (the “Company”), a genomic medicine company developing vision saving treatments for ocular diseases, today announces the appointment of Dr Daniel C. Chung as Chief Medical Officer (“CMO”). Dr Chung brings a wealth of experience in successful ocular gene therapy development, having led the global medical strategy for ophthalmology at Spark Therapeutics (“Spark”), a leading rare disease gene therapy company, where he played a pivotal role in the clinical development and approval of Luxturna™, the first gene therapy to be approved in the US for a genetic disease. Spark was acquired by Roche in 2019 for $4.3 billion.

Dr Chung is a highly respected healthcare leader with a demonstrated history working in the biotechnology industry, with experience spanning all phases of ocular gene therapy development, from pre-clinical studies through to clinical development and post marketing activities. Dr Chung joined Spark in 2014 and, as the company’s first ophthalmologist, was instrumental in the development of Luxturna™, the first in vivo gene therapy approved by the US Food and Drug Administration (“FDA”) for RPE65 related inherited retinal disease. He also later led the medical affairs group responsible for its safe and efficient use in patients. During his tenure at Spark, he served as the inherited retinal disease and ophthalmology expert across various company divisions, not only training preclinical staff for in vivo surgical procedures, but also working in medical affairs, clinical development, patient advocacy, marketing and commercial activities.

Before joining Spark, Dr Chung worked for 11 years at the Scheie Eye Institute within the Perelman School of Medicine at the University of Pennsylvania, specialising in gene based therapies for inherited retinal diseases and cilia mediated disease. Dr Chung received his medical degree from the New York College of Osteopathic Medicine in 1994 and undertook extensive postgraduate training at the National Eye Institute, Summa Health Systems and Cole Eye Institute at the Cleveland Clinic Foundation.

As a member of the executive team at SparingVision, Dr Chung will lead the clinical development and research of SPVN06, SparingVision’s flagship development asset, that is uniquely positioned to treat a much wider population of patients with rod-cone dystrophies than existing gene therapies. He will also be responsible for the clinical development of future additions to the product pipeline. Based in the U.S., Dr. Chung will also lead the education of the medical and patient communities on SparingVision’s unique and game-changing mutation-agnostic approach in gene therapy.

Stephane Boissel, SparingVision President and Chief Executive Officer, said, “SparingVision is focused on leading a significant step shift in the way we treat ocular disease, and we are building a strong global team with the expertise and drive to deliver this vision. We are delighted to have attracted another key leader in the field, which is testament to the potential of our approach. Dan’s extensive ophthalmology knowledge and experience in successfully bringing an ocular gene therapy to market will be highly valued as we prepare to commence our first-in-human study of SPVN06 and investigate the further potential of this asset in other rod-cone dystrophies.”

Dr Daniel C. Chung, Chief Medical Officer of SparingVision, added, “SparingVision’s pioneering science and the team’s track record in drug development, manufacturing and building of successful companies is impressive. SPVN06 could allow the treatment of rod-cone dystrophies to go beyond single gene correction approaches to enable central vision preservation for patients, regardless of genetic cause. I am looking forward to joining SparingVision at such an exciting time in the development of this approach and potentially changing the lives of many patients who currently have no approved treatment options.”

 

About SparingVision:
SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

About SPVN06:
SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVision’s primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2021.

Contacts:
SparingVision
Stéphane Boissel
President and CEO

Nathalie Trepo
Investor Relations
nathalie.trepo@sparingvision.com


Consilium Strategic Communications
Amber Fennell, Olivia Manser, Lizzie Seeley
+44 (0)20 3709 5700
sparingvision@consilium-comms.com

Development of lead asset SPVN06 to further benefit from high-level Clinical Advisory Board

Paris, January 29, 2021 – SparingVision (the “Company”), a genomic medicine company developing vision saving treatments for ocular diseases, today announces the strengthening of its Scientific Advisory Board (“SAB”) with the appointment of internationally renowned ophthalmology pioneers Dr. Paul A. Sieving, Professor of University of California Davis School of Medicine and the former Director of the US National Eye Institute, and Prof. Botond Roska, co-director of the Institute of Molecular and Clinical Ophthalmology Basel and Professor at the Medical and Science Faculty of the University of Basel.

Prof. José Alain Sahel, co-founder of SparingVision, said, “To have international scientists of this calibre joining our SAB is further validation of the potential of the Company’s science, based on decades of work at the Paris Vision Institute. We will benefit tremendously from the knowledge, advice and insights of these ophthalmology leaders, which will aid us in our mission to bring next generation genomic medicines to patients with blinding ocular diseases as quickly as possible.”

Stéphane Boissel, President and Chief Executive Officer of SparingVision, added, “The addition of these renowned scientists to our SAB is perfectly timed as we prepare to commence our first in human study of SPVN06 for the treatment of retinitis pigmentosa later this year. Moreover, as we look to build a coherent portfolio of genomic medicines in the field of ophthalmology, their advice will be invaluable.”

Dr. Paul A. Sieving is an ophthalmologist with extensive expertise in inherited retinal diseases (“IRD”). He served as Director of the National Eye Institute at the National Institutes of Health for nearly 20 years where he led the research programs to advance national vision health. Dr. Sieving previously was the Paul R. Lichter Professor of Ophthalmic Genetics at the University of Michigan Medical School for 17 years and founded the Centre for Retinal and Macular Degeneration. Dr. Sieving received his M.D. from the University of Illinois where he trained in ophthalmology and obtained a Ph.D. in bioengineering. He has developed and led translational therapy programs for IRD conditions using human ocular gene therapy and neurotrophic factor implants. Dr. Sieving has received prestigious awards including the Società Oftalmologica Italiana Honorary Award in Ophthalmology in 2016. He holds elected membership in the National Academy of Medicine USA and also in the German National Academy of Science. Since leaving the NIH in 2019, he founded the new Centre for Ocular Regenerative Therapy at the University of California, Davis.

Prof. Botond Roska is co-founder of the Institute of Molecular and Clinical Ophthalmology Basel (“IOB”), which is dedicated to advancing the understanding of vision, its diseases and to developing new therapies for vision loss. Prof. Roska received his PhD in Neuroscience at the University of California, Berkeley. He then studied genetics and virology as a Harvard Society Fellow at Harvard University and Harvard Medical School. He has been a professor at the Medical Faculty of the University of Basel since 2014 and professor at the Faculty of Science since 2019. Prof. Roska has received numerous awards for his outstanding accomplishments in vision science research, including the 2020 Körber European Science Prize, awarded for his pioneering research over the last 20 years exploring mechanisms of vision, how different cells in the visual system extract information from the environment and the complex interplay of information involved in signal processing. His research is currently focused on gene therapies that restore light sensitivity to retinal cells to renew functionality in blind retinas.

Dr. Sieving and Prof. Roska join existing SAB members, led by SparingVision’s co-founder Prof. José Alain Sahel, a key figure in the field of vision restoration and eye regenerative therapies; Prof. Jean Bennett, Co-Director of Centre for Advanced Retinal & Ocular Therapeutics at University of Pennsylvania and the driving force leading to the approval of Luxturna®; Dr. Elias Arnér, Head of the Biochemistry Division in the Department of Medical Biochemistry and Biophysics at Karolinska Institutet, Stockholm, and Prof. Pierre Chambon, Professor of Molecular Genetics at the Institute of Advanced Studies Strasbourg University Founder & former Director of the Institute for Genetics, Cellular and Molecular Biology, France.

With great sadness SparingVision acknowledges the passing of Dr. Arne Holmgren, who was a member of the SAB since the onset. Dr. Holmgren was a Senior Professor at the Karolinska Institutet and widely known as one of the ground-breaking scientists establishing the rapidly growing field of redox biology. The Company is truly grateful for his advice over the years and offers its deepest condolences to his family.

SparingVision’s SAB will counsel the Company on the scientific development of its research program as well as the strategic expansion of its portfolio. The clinical development of SPVN06, a proprietary, mutationagnostic, AAV gene therapy and the Company’s lead asset, will further benefit from the support of SparingVision’s Clinical Advisory Board (“CAB”) comprised of the following seven leaders in the field of gene therapy and ophthalmology:

  • Prof. Jean Bennet, Co-Director of Centre for Advanced Retinal & Ocular Therapeutics at University of Pennsylvania;
  • Prof. Jacque Duncan, Academic Director, Retina Service, Department of Ophthalmology, UCSF and Chair of Foundation Fighting Blindness’s Scientific Advisory Board;
  • Prof. Eric Pierce, Director of the Ocular Genomics Institute and Professor at Harvard Medical School;
  • Prof. Eyal Banin, Director of Centre for Retina at Hadassah Medical Centre and professor at the Hebrew University, in Jerusalem;
  • Prof. Mark Pennesi Ophthalmic Genetics Associate Professor at Oregon Health & Science University;
  • Prof. Bart Leroy, Head of Department of Ophthalmology, Ghent University Hospital, Belgium and Professor of Ophthalmology at the University of Pennsylvania;
  • Prof. Masayo Takahashi, stem cell trailblazer and a former project research leader at the Riken Centre for Developmental Biology in Japan and the current President of VisionCare.

About SparingVision:
SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised €60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.

About SPVN06:
SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVision’s primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in 2021.

Contacts:
SparingVision
Stéphane Boissel
President and CEO

Nathalie Trepo
Investor Relations
nathalie.trepo@sparingvision.com


Consilium Strategic Communications
Amber Fennell, Olivia Manser, Lizzie Seeley
+44 (0)20 3709 5700
sparingvision@consilium-comms.com

Round was led by Sofinnova Investments with participation from Abingworth, Lightstone Ventures and all existing investors

Company expands board of directors and plans to build out team

DURHAM, NC and BOSTON, MA – December 16, 2020 – Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced it has closed an oversubscribed $55 million Series A financing led by Sofinnova Investments with participation from additional new investors Abingworth and Lightstone Ventures. Founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund, along with existing investors Osage University Partners, University of Florida, and Manning Family Foundation, also participated in the round. Sarah Bhagat, PhD, Partner at Sofinnova, Jackie Grant, PhD, Principal at Abingworth, and Jason Lettmann, General Partner at Lightstone, will join Atsena’s board of directors.

Proceeds will be used to advance Atsena’s ongoing Phase I/II clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children, as well as complete manufacturing development for Phase 3. In addition, the funds will enable Atsena to expand its team to support the research and development of novel gene therapies, including the progression of two existing preclinical programs in inherited retinal diseases toward the clinic and advancement of the company’s innovative adeno-associated virus (AAV) technology platform.

“We are grateful for the support of our new and existing investors and are encouraged by their enthusiasm for the potential of our technology to overcome the unique hurdles of inherited retinal diseases to prevent or reverse blindness,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. “The Series A financing provides financial runway to reach the key inflection point of reading out efficacy data from our LCA1 clinical trial. While we continue expeditiously advancing this trial and our preclinical programs, we are excited to accelerate our growth as a leading ophthalmic gene therapy company.”

The Phase I/II LCA1 clinical trial is currently enrolling patients in the second dosing cohort. Atsena exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida. LCA is the most common cause of blindness in children. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.

“We believe Atsena’s foundation in ocular gene therapy and potentially game-changing novel AAV vectors position the company to become a partner of choice,” said Dr. Bhagat. “Sofinnova is delighted to support Atsena and we look forward to helping the team further its mission to develop life-changing gene therapies for patients with inherited retinal diseases.”

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and has offices in Boston, MA and North Carolina’s Research Triangle, environments rich in gene therapy expertise. For more information, please visit atsenatx.com

About Sofinnova Investments
Since our founding in 1974, Sofinnova has been active in life science investing. We are a clinical-stage biopharmaceutical investment firm with approximately $2.3B in assets under management and committed capital. We invest in both private and public equity of therapeutics-focused companies. Our goal is to actively partner with entrepreneurs in both the U.S. and Europe, across all stages of company formation. From drug development and navigating the regulatory process to company building and IPO, we strive to be collaborative, meaningful board members, and excellent partners at every level. We seek to build world class companies that aspire to dramatically improve the current state of medical care today and ultimately, the lives of patients. Sofinnova has expertise investing in gene therapy companies, including investments in Spark, which developed the first approved gene therapy, Akouos, and Audentes, and Xylocor. For more information, please visit www.sofinnova.com.   

About Abingworth
Abingworth is a leading transatlantic life sciences investment firm. Abingworth helps transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams building world-class companies. Since 1973, Abingworth has invested in approximately 168 life science companies, leading to more than 44 M&A/exits and close to 70 IPOs. Our therapeutic focused investments fall into 3 categories: seed and early-stage, development stage, and clinical co-development. Abingworth supports its portfolio companies with a team of experienced professionals at offices in London, Menlo Park (California) and Boston. For more information, visit abingworth.com.  

About Lightstone Ventures
Lightstone Ventures is a leading venture capital firm investing in therapeutic-oriented companies across the life science spectrum, from breakthrough medical devices to novel drugs and biopharmaceuticals. Founded in 2012, Lightstone has been part of many successful new ventures from inception through commercialization and plays a critical role guiding and building successful healthcare companies. With a proven strategy and global footprint, the Lightstone team has been involved in several of the largest venture-backed life science exits over the last decade including: ALX Oncology, Acceleron, Ardian, Calithera, Claret Medical, Disarm, MicroVention, Nimbus, Plexxikon, Portola, Promedior, Proteolix, Ra Pharma, Tizona, Twelve and Zeltiq. For more information, visit https://www.lightstonevc.com

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
tplohoros@6degreespr.com

Business Contact:
info@atsenatx.com

Stargazer Pharmaceuticals, Inc. Announces $57 Million Series A Financing and Initiation of a Phase 2a Clinical Study of STG-001 in Stargardt Disease Patients

Boston, MA; November 9, 2020

Stargazer Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of novel treatment options for rare eye diseases, announced it has completed a Series A financing totalling $57 million from leading investors Novo Holdings, venBio Partners, Canaan Partners and Pontifax to develop its proprietary compound STG-001 for treating Stargardt Disease.  Stargazer also announced the initiation of a Phase 2a study of STG-001 in Stargardt Disease patients.

This Series A financing enables Stargazer to advance development of STG-001 through a pivotal clinical efficacy study potentially leading to a new therapy for Stargardt Disease, a major cause of vision loss in children and young adults.  Stargazer is pleased to be collaborating with Foundation Fighting Blindness (FFB) for the development of STG-001. FFB also participated in this financing round via their investment vehicle, the Retinal Degeneration Fund.

Stargazer was launched by Novo Ventures, the venture arm of Novo Holdings, Gary Sternberg, MD, MBA, who serves as the company’s Chief Executive Officer, and Silvia Ragno, PhD, who serves as the company’s Chief Operating Officer, following licensing of STG-001 from Takeda Pharmaceuticals.

Concurrent with the financing, Richard Gaster, MD, PhD, a Partner at venBio Partners, and Wende Hutton, a General Partner at Canaan Partners, will join Stargazer’s Board of Directors.  They join Nanna Lüneborg, a Partner at Novo Ventures, and Iyona Rajkomar, a Partner at Pontifax.  Other Board members include Daniel J. Curran, MD, from Takeda Pharmaceuticals, John Wagner, MD, PhD, and Dr. Sternberg.

Stargardt Disease is a genetic orphan disease that leads to legal blindness.  It is the most common juvenile macular dystrophy affecting 1 in 8,000 to 1 in 10,000 individuals.  There are currently no therapies available for this disease.  The Phase 2a study of STG-001 is a multi-center study in the US of subjects with Stargardt Disease comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.*

Stargazer has successfully completed a Phase 1 single- and multiple-ascending dose clinical study of STG-001 in healthy volunteers and is planning to initiate a pivotal efficacy study in 2021.

STG-001 has received Orphan Drug Designation in both the US (Food and Drug Administration) and EU (European Medicines Agency).

“This financing from leading investors, as well as the support from FFB, is a testament to the potential of our lead compound, STG-001.  Initiation of the Phase 2a trial in Stargardt Disease patients is an important milestone for us and we look forward to further development of STG-001 for the benefit of Stargardt Disease patients,” said Dr. Gary Sternberg. 

“We are very excited to work with Stargazer to develop a novel and potentially best in class therapy for the treatment of Stargardt Disease, a disease with a high unmet medical need,” said Nanna Lüneborg of Novo Ventures.

About STG-001

The visual cycle is fuelled by uptake of Vitamin A (retinol) from the circulation that is transported by Retinol Binding Protein 4 (RBP4), a key protein that binds retinol in the systemic circulation and delivers it to the eye.

STG-001 is an indirect visual cycle modulator. By reducing plasma concentrations of RBP4 and Vitamin A, STG-001 modulates the visual cycle and the accumulation of cytotoxic retinoids in the eye, potentially reducing the rate of damage to the retina and preserving vision in Stargardt Disease patients.

Stargardt Disease

Stargardt Disease is an inherited retinal degenerative disease that leads to progressive central visual loss and legal blindness.  It is the leading cause of childhood blindness with an estimated prevalence of 50,000-80,000 patients in the US and major European countries combined.  It occurs in 1 in 8,000 to 1 in 10,000 people making it the most common juvenile macular dystrophy.

There is no treatment currently available.  Patients typically exhibit comorbidities, including depression.  Typical onset of disease is generally between 10-20 years of age. 

About Stargazer Pharmaceuticals, Inc.

Stargazer Pharmaceuticals, Inc., is a Boston-based biopharmaceutical drug development company launched in 2018 with a focus on rare diseases of the eye, and an initial focus on Stargardt Disease.  For more information, please visit https://www.stargazerpharmaceuticals.com/

* https://clinicaltrials.gov/ct2/show/NCT04489511?term=stg-001&draw=2&rank=1) 

 

 

 

 

  • Acquisition of Vedere Bio includes lead preclinical intravitreally-injected AAV gene therapy programs focused on pan-genotypic vision restoration in patients with photoreceptor-based vision loss.
  • $150 million upfront with the remainder in early regulatory and clinical milestones for a total of $280 million.
  • Newly formed Vedere Bio II will leverage an ocular gene therapy toolbox to develop a new pipeline of novel vision restoration and vision preservation therapies.

Vedere Bio, Inc. (Vedere Bio), a stealth-stage company focused on advancing photoreceptor-protein-based optogenetic therapies that are delivered to the retina intravitreally to restore functional vision, announced today that it has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and will be eligible for up to $130 million in milestone payments, for a total of $280 million. Based on technology from the laboratories of Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, and technology directed at enhanced ocular gene therapy delivery arising jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania, Vedere Bio was formed in the Atlas Venture incubator in June 2019. The company was launched with a $21 million Series A financing and began lab operations at LabCentral in Cambridge, MA where it advanced its lead programs from concept to development candidate within one year. Immediately prior to the acquisition, certain earlier-stage vision restoration and vision preservation assets leveraging the company’s ocular gene therapy toolbox were spun out into a newly formed entity – Vedere Bio II, Inc.

“The medical need for new therapies to treat blindness is unambiguous,” said Jay Bradner, President of the Novartis Institutes for BioMedical Research. “Vedere Bio’s innovative technologies expand the potential for gene therapy to improve the lives of patients facing vision loss due to photoreceptor death attributable to a number of prevalent eye diseases.”

“Vedere Bio’s photoreceptor-protein-based optogenetics program has important advantages over competing approaches and brings us one step closer to delivering functional vision to patients in need.  Our proprietary intravitreal capsids enable not only Vedere Bio’s optogenetics products but also other ocular gene therapies,” said Cyrus Mozayeni, M.D., Chief Executive Officer, President of Vedere Bio and Atlas Venture Entrepreneur in Residence. “Our sale to Novartis is an important milestone in advancing Vedere Bio’s most advanced programs to patients around the world. At the same time, I look forward to working with our experienced team to advance our highly innovative, earlier stage assets as part of the newly established Vedere Bio II.”

“Through Vedere Bio’s approach, we are able to see the impact of venture philanthropy in achieving real progress in the development of new therapies for patients with vision loss,” said Ben Yerxa, Ph.D., CEO of Foundation Fighting Blindness. “For the millions of patients globally living with inherited retinal degenerations and other eye diseases caused by photoreceptor death, the advancement of novel optogenetics therapies provides hope for the future. In addition, we are eager to partner with Vedere Bio II to accelerate their novel ocular gene therapy programs.”

The newly formed Vedere Bio II, Inc. will operate as a wholly independent entity from Novartis and Vedere Bio. Vedere Bio II aims to develop a pipeline of novel vision restoration and vision preservation medicines by targeting underserved indications. Backed by the full Vedere Bio investor syndicate of Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund), the Vedere Bio II team will launch with Vedere Bio’s founders, team and facilities to advance its pipeline.

 “The acquisition of Vedere Bio by Novartis speaks to the strength of the underlying science from our founders and to the incredible job the team has done in advancing these programs over the past year. We are very excited by the potential of this technology and the opportunity to deliver true restoration of sight for patients who have been living in darkness for so long,” said Kevin Bitterman, Ph.D., Partner at Atlas Venture and Chairman of the Vedere Bio Board of Directors. “Since Vedere Bio was founded in 2019, the company has moved with unprecedented urgency over the course of one year to advance from concept to development candidate. We look forward to the advancement of the lead programs by Novartis and are eager to see the progress of the Vedere Bio II team in the future to develop novel ocular gene therapies for patients in need.”

The transaction closed in September 2020.

About Vedere Bio II, Inc.

Vedere Bio II, which will be referred to as Vedere going forward, is a privately held, emerging biopharmaceutical company leveraging an ocular gene therapy toolbox to develop a pipeline of vision restoration and vision preservation therapies. Comprised of a diverse team of pioneering scientists, Vedere Bio II is discovering and developing next generation ocular gene therapies to make vision restoration a reality in areas of high global unmet medical need. The company is headquartered in Cambridge, MA and is funded by Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund). For more information, please visit www.vederebio.com or follow Vedere Bio II on Twitter and LinkedIn.

Paris, October 21, 2020 – SparingVision (“the Company”), a genomic medicine company focused on ocular diseases, today announces a €44.5 million financing round. The round was led by 4BIO Capital (“4BIO”) and UPMC Enterprises, and included Jeito Capital (“Jeito”) and Ysios Capital (“Ysios”). In addition, current investors Bpifrance and Foundation Fighting Blindness (“FFB”) participated in the round.

Proceeds from the financing will be primarily used to advance the development of SparingVision’s
breakthrough treatment SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa (“RP”). Most
notably the funding will support SparingVision’s GMP activities (including the manufacturing of a first clinical
batch of the product, currently ongoing), the IND/CTA regulatory activities and the conduct of a first-in-man
study, scheduled to start in 2021. The Company also intends to further expand its management team and
commence operations in the US.

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one
oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of
photoreceptors. Loss of photoreceptors leads to blindness in RP, one of the most common inherited retinal
diseases that affects two million patients worldwide. There is currently no treatment approved for RP
patients independently of their genetic background.

Torreya Capital, LLC served as exclusive placement agent for the offering.

SparingVision also announces today that the Company’s Chairman of the Board, Stéphane Boissel, has been
appointed Chief Executive Officer of the Company.

Stéphane Boissel, SparingVision’s President and Chief Executive Officer, said,We are delighted to have closed this financing round, which demonstrates the excitement around SparingVision’s lead compound, SPVN06. With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals.

Before joining SparingVision as President and CEO, Stéphane Boissel was Executive Vice President,
Corporate Strategy at Sangamo Therapeutics (“Sangamo”), a Nasdaq-listed gene-editing company based in
San Francisco, California. Prior to Sangamo, he was CEO of TxCell, the first ever CAR-TReg company, that
was sold to Sangamo in 2018. Prior to TxCell, Stéphane served as CEO of Genclis, a molecular diagnostic
company, and EVP and CFO of Innate Pharma, a Nasdaq-listed company, and Transgene.

Stéphane has also been a Board member of several leading biotechnology companies, including Nasdaqlisted Erytech Pharma and Elsalys Biotech, where he served as Chairman. Earlier in his career, Stéphane worked in investment banking for Lazard, where he focused on principal investment in France, Singapore and Hong Kong. He started his career at PWC. Stéphane graduated in management and finance from the IAE Lyon, University of Lyon and PSL Paris-Dauphine University (France) and received his MBA from the University of Chicago Booth School of Business.

Dr. José-Alain Sahel, Chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine, Director of the Institut de la Vision (Sorbonne Universite, Inserm, CNRS, Paris) and co-founder of SparingVision along with Dr. Thierry Léveillard, added, “SparingVision’s neuroprotective approach, which I started working on some 20 years ago at Institut de la Vision, has all the attributes of a winning gene therapy solution for retinitis pigmentosa patients in desperate need of a universal therapeutic option. I am looking forward to helping Stéphane and the team best position SPVN06 for clinical development up to regulatory licensing.

The Board of Directors of SparingVision expresses its deepest gratitude to Florence Allouche, co-founder, who is leaving her position as President of the Company, for her hard work and support over the years which has enabled the Company to pass many milestones and successfully refinance itself.

DURHAM, N.C., July 29, 2020 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that it has acquired exclusive rights to a gene therapy targeting GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina and is a leading cause of blindness in children, from Sanofi, which originally licensed it from the University of Florida. The therapy was created in the laboratory of Atsena Founder and Chief Scientific Officer Shannon Boye, Ph.D., and Founder and Chief Technology Officer Sanford Boye, M.Sc., at the University of Florida.

“We are thrilled that our gene therapy for LCA1 is coming home to Atsena and that we will have the opportunity to further its development,” said Shannon Boye. “Atsena was founded to advance treatments for inherited retinal diseases and believes in centering patients’ perspectives and needs in all we do. We are honored to continue to work with LCA1 patients and their families as we strive to treat this debilitating disease.”

LCA is the most common cause of blindness in children, impacting two to three per 100,000. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.

Atsena has an ongoing Phase I/II clinical trial evaluating this gene therapy in LCA1 patients. The second cohort in the trial is expected to be dosed in the fall of 2020.

“Atsena’s gene therapy has the potential to be a major advance in treating blindness in both children and adults affected by this inherited retinal disease,” said Benjamin Yerxa, Ph.D., Chief Executive Officer of the Foundation Fighting Blindness and Atsena board director. “The foundation was instrumental in supporting proof of concept studies in the founders’ labs over the last 15 years. Now, via investment in Atsena through our Retinal Degeneration (RD) Fund, we are excited to support this potential breakthrough treatment for LCA1.”

Atsena closed a Series 1 funding of $8.15 million in April 2020, led by founding investors Hatteras Venture Partners and the Foundation Fighting Blindness’ RD Fund with participation by Osage University Partners, PBM Capital and the University of Florida. Patrick Ritschel, M.B.A., co-founder and former President of gene therapy company StrideBio, serves as Atsena’s Chief Executive Officer.

“Atsena is pleased to have the support of an enthusiastic investor base that shares our dedication to bringing the life-changing power of genetic medicine to patients living with LCA1 and other forms of blindness,” said Ritschel. “We look forward to working closely with our investors and patients as we continue to grow, and expect to announce additional milestones later this year.”

About Atsena Therapeutics

Atsena Therapeutics is a clinical-stage gene therapy company, focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. The company has an ongoing Phase I/II clinical trial evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome the hurdles presented by inherited retinal disease, and its approach is guided by the specific needs of each patient condition. Founded by pioneers in ocular gene therapy, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and is headquartered in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com.

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
tplohoros@6degreespr.com

Nacuity Pharmaceuticals Announces Initiation of SLO-RP (Safety and Efficacy of NPI-001 Tablets versus PLacebO for Treatment of Retinitis Pigmentosa Associated with Usher Syndrome) Phase 1/2 Clinical Trial 

NPI-001 Being Evaluated for the Treatment of Retinitis Pigmentosa in Patients with Usher Syndrome

FORT WORTH, TX, May 18, 2020 – Nacuity Pharmaceuticals, Inc., a clinical stage biotech company focused on the treatment of ocular diseases involving oxidative stress, today announced the initiation of a Phase 1/2 study of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH). The study, named SLO-RP, is designed to demonstrate greater retinal sensitivity in RP/USH patients treated with NPI-001 Tablets versus placebo. Previously, NPI-001 was evaluated in a Phase 1 study conducted in healthy volunteers, demonstrating its safety profile. The SLO-RP study will enroll patients diagnosed with RP associated with USH who are early in disease progression. 

Preclinical studies indicate that oral NPI-001 protects photoreceptor cones in the eye from oxidative damage, which may preserve visual function.1 Furthermore, NPI-001 and related small molecules have been shown to be potent antioxidants in several preclinical models that may lead to additional indications.2 “Initiation of the SLO-RP trial is an important milestone in our efforts to understand the potential of this treatment for RP and a prelude to treating other conditions of the eye involving oxidative stress,” said G. Michael Wall, PhD, senior vice-president and chief scientific officer at Nacuity. 

Nacuity Pharmaceuticals has partnered with the Foundation Fighting Blindness on a variety of initiatives related to the development of NPI-001 including funding through the Foundation’s venture arm the RD Fund, trial design, awareness and recruitment, and additional strategic guidance. “We are happy on behalf of the many patients around the globe who are affected by RP to see this promising treatment reach the clinic.” said Dr. Ben Yerxa, chief executive officer of Foundation Fighting Blindness, “We remain committed to seeing this potential treatment through to the next milestone.” 

About the SLO-RP Trial

The randomized, placebo-controlled, multicenter, double-masked, dose escalation trial is designed to assess the clinical safety, tolerability and efficacy of NPI-001 Tablets versus placebo in patients with USH (NCT04355689). The Australian study will target enrollment of at least 48 male and female USH patients, ages 18 years and older, who will be followed for 2 years. The goals are to assess the safety of chronic dosing up to 500mg/day and generate clinical proof of concept for the treatment of RP. The key clinical endpoint is change from baseline in retinal sensitivity of patients treated with NPI-001 versus placebo. 

Retinitis Pigmentosa

RP refers to a group of inherited diseases causing retinal degeneration and vision loss that worsen over time including USH, Leber congenital amaurosis, rod-cone disease, Bardet-Biedl syndrome, Refsumdisease, and others. The vision loss in USH is due to RP, presenting in the same manner.  By showing a slowing of disease progression in the SLO-RP study, Nacuity aims to begin to address RP as a whole.

The National Eye Institute (NEI) estimates that approximately 100,000 people in the United States have RP. About 1.5 million people are afflicted worldwide. Night blindness is one of the earliest and most frequent symptoms. RP is a progressive disorder typically diagnosed in adolescents and young adults. The rate of progression and degree of visual loss varies by case, with the majority of patients legally blind by age 40. The only FDA-approved treatment for RP is LUXTURNA®, a gene therapy applicable for about 1% of RP patients (rpe65). (Trademarks are property of their owners.)

Nacuity Pharmaceuticals, Inc. (https://www.nacuity.com) is a clinical stage biotech company focused on advancing treatments for ocular conditions involving oxidative stress. Nacuity is funded by Foundation Fighting Blindness grants and direct investment, as well as private investors.  

Foundation Fighting Blindness (https://www.fightingblindness.org

Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $760 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit FightingBlindness.org for more information.

Forward Looking Statements

This announcement includes "forward-looking statements" within the meaning the federal securities laws. All statements other than statements of historical facts contained in this press release, including statements regarding our anticipated future clinical and regulatory events, future financial position, business strategy and plans and objectives of management for future operations, are forward-looking statements. Forward looking statements are generally delivered in the future tense and/or are preceded by words such as "may," "will," "should," "forecast,“ "projected," "could," "expect," "suggest,”

"believe," "estimate," "anticipate," "intend," "plan,“ or similar words, or the negatives of such terms or other variations on such terms or comparable terminology. Such forward-looking statements include, without limitation, statements regarding the potential future commercialization of our product candidates, the anticipated start dates, durations and completion dates, as well as the potential future results of our future clinical trials, the anticipated designs of our future clinical trials, anticipated future regulatory submissions and events, our anticipated future cash position and future events under our current and potential future collaborations. 

1Dong A, Stevens R, Hackett S, Campochiaro PA. Compared with N-acetylcysteine (NAC), N-Acetylcysteine Amide (NACA) Provides Increased Protection of Cone Function in a Model of Retinitis Pigmentosa. Investigative Ophthalmology & Visual Science April 2014, Vol.55, 1736.

2Nacuity data on file.

CheckedUp Launches Virtual Visits, Advanced Telehealth Platform for Specialty Care

Advanced Telemedicine System Delivers Patients, Physicians, and Partners an Exceptional Virtual Care Experience that Redefines Telemedicine

April 28, 2020 09:27 AM Eastern Daylight Time

NEW YORK--(BUSINESS WIRE)--CheckedUp – the fastest growing specialty medicine digital point of care company – today announced the launch of CheckedUp Virtual Visits, an innovative, advanced telemedicine system designed to redefine virtual specialty care.

CheckedUp Virtual Visits, designed by physicians for physicians, was created to keep patients and physicians at the center of pivotal healthcare decisions as “point of care” engagement evolves with patient needs and becomes increasingly digital. As the COVID-19 pandemic continues to alter the world’s “new normal,” CheckedUp is proud to offer a valuable, innovative service that redefines the virtual consultation experience. Its innovative platform empowers specialty care physicians, patients, and industry partners in multiple, dynamic ways.

“From early on, our leadership team viewed the COVID-19 crisis as an unprecedented challenge requiring an innovative response,” said Mark Goethals, Vice President of Marketing, CheckedUp. “Our efforts are focused on a new, post-COVID healthcare delivery landscape, delivering the service and functionality of a virtual examination room to patients and enabling providers to deliver superior virtual patient care. CheckedUp Virtual Visits allows physicians to meet their patients when and where they are, while providing innovative tools for providers and patients during their digital consultation. We are also happy to extend that accessibility to our partners in the life sciences, so that they can be a part of tomorrow’s healthcare conversations.”

CheckedUp Virtual Visits offers distinct care advantages over traditional telemedicine systems. It simplifies scheduling and operations, allows patients to see the dedicated specialist they know and trust, and brings the same easy to use technologies physicians use in the physical exam room, into the virtual realm.

“CheckedUp Virtual Visits leads a new era in the way physicians engage with patients during virtual consultations,” said Dan Schwartz, Senior Vice President, Sponsorship Sales, CheckedUp. “Current telemedicine systems, while serving an important role today, offer very little by way of additional in-office technologies, making exceptional care possible. We expect to change that.”

CheckedUp Virtual Visits is designed to perfectly replicate the patient and physician experience in a virtual setting, streamline administrative efforts by tracking physicians' personal notes and information regarding every consult, and remain HIPAA Compliant.

About CheckedUp

CheckedUp is one of New York’s fastest growing healthcare technology growth companies and is the only 100% digital, push technology platform designed for specialty point of care. CheckedUp has deployed a state-of-the-art platform into Specialty Healthcare facilities across the United States to actively engage patients, caregivers, and physicians in the waiting room, exam room, and at home. As a healthcare technology leader, CheckedUp aspires to create better educated and more confident patients and physicians who are empowered to make better health decisions together. CheckedUp was ranked #279 on Inc. 5,000’s list of the Fastest Growing Private Companies in the US. Learn more at www.CheckedUp.com.

 

ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update

  • QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events
  • QR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platform
  • COVID-19 pandemic expected to impact timelines for the pipeline
  • ProQR anticipates its cash runway will fund operations into H2 2022

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 31, 2020 (GLOBE NEWSWIRE) --  March 31, 2020 -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announces positive findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. The Company is also providing an update on business operations in relation to the COVID-19 pandemic. Management will host a conference call today at 8:15 am ET.

“The goal of the interim analysis of this 24 month Stellar trial of QR-421a was to assess safety and early signs of efficacy for the purpose of informing next steps in development and future trial strategy,” said David Rodman, M.D., Executive Vice President of Research and Development of ProQR. “We are pleased with the current safety profile and are very encouraged by early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% of QR-421a-treated patients thus far in this trial. The findings support continuing the trial as planned, with both cohort expansion and dose escalation in order to identify a potential development path to registration. Importantly, these data represent the second program from our ophthalmology pipeline that is supported by preclinical predictions from human retinal organoids, providing further validation of our translational approach and platform technology.”

Phase 1/2 Three-Month Interim Analysis

The interim analysis (IA) is based on nine and three month data from the first and second dose  cohorts, respectively, of the Stellar Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy. The Stellar trial is a randomized, single ascending dose, global multicenter, longitudinal, 24-month study, involving active versus sham procedure. The first two cohorts include a total of 14 subjects (ranging from 24-65 years in age), of which eight received a single dose of QR-421a and six received a single sham procedure for masking. Six subjects were enrolled in the 50 µg cohort (“low dose”), of which four received treatment and two were randomized to sham; eight patients were enrolled in the 100 µg cohort (“mid dose”) of which four received treatment and four were randomized to sham. The population varied in disease characteristics with both Usher syndrome (n=6) and nsRP (n=8) affected subjects included, genetic background with both homozygous (n= 4) and heterozygous (n=10) subjects for USH2A exon 13 mutations, and visual impairment at baseline ranging from mild to severe.

Key initial findings include the following:

Safety data: Across both cohorts thus far, QR-421a was observed to be generally well tolerated with no serious adverse events noted.

Efficacy data: In the six sham treated subjects (two followed for 9 months and four for 3 months), outcome measures demonstrated no consistent pattern of response above the “noise” level.  In contrast, two of eight QR‑421a-treated patients (one each in the 50 µg and 100 µg dose cohorts) demonstrated benefit across multiple concordant outcome measures.  

  • Responder 1: One of four treated patients in the low dose group was classified as a responder, with onset of action observed by the 3 month visit. Benefit was maintained for 6 months or longer, which is consistent with the expected half-life of QR-421a in photoreceptors. This Usher syndrome patient was homozygous for USH2A exon 13 mutations and had moderate visual impairment at baseline (peripheral vision affected). Concordant benefit was observed across multiple relevant measures appropriate to the severity of the patient’s disease, including full field stimulus threshold test (FST) [deterioration by 5 dB in untreated eye, treated eye stable], dark adapted chromatic (DAC) perimetry [15 dB.steradian improvement in peripheral sensitivity in treated eye, <5 dB.steradian change in untreated eye], and optical coherence tomography (OCT) assessment of photoreceptor Ellipsoid Zone (EZ area). For FST and OCT, the contralateral, untreated eye demonstrated modest deterioration while the treated eye showed stabilization. For DAC perimetry the untreated eye was unchanged, whereas the treated eye demonstrated improvement.
  • Responder 2: One of four treated patients in the mid dose group was classified as a responder with onset of action observed by 3 months. This non-syndromic RP patient was heterozygous for USH2A exon 13 mutations and had severe visual impairment at baseline (peripheral and central vision affected) with baseline best corrected visual acuity (BCVA) of 33 and 36 letters (approximate Snellen equivalent: 20/250 and 20/200) in the treated and untreated eye, respectively. Concordant benefit was observed across multiple relevant measures appropriate for the stage of disease including FST (improvement by 12 dB in treated eye, no improvement in untreated eye), DAC (up to 10 dB.steradian improvement in treated eye, with deterioration in the untreated eye), and BCVA (7 letter improvement from baseline of 33 letters, which is more than one line on the ETDRS eye chart, compared to no change in the untreated eye).

Next steps: Based on the safety profile and early evidence of efficacy observed to date, the Company plans to take advantage of the adaptive design, and expand the 100 µg cohort with additional subjects who are homozygous for exon 13 mutations. Dose escalation to 200 µg (“high dose”) is planned to occur in parallel. An interim analysis of dose- and gene copy-dependent safety and efficacy will be planned once all additional subjects have reached at least 3 months of treatment.

Business Update Related to COVID-19 Pandemic

The COVID-19 pandemic is rapidly evolving and has prompted global health concerns, the duration, severity and exact impact of which are currently unknown and cannot be predicted with confidence. In consultation with the trial sites, due to the COVID-19 pandemic the Company also expects a delay in all of its ongoing and scheduled trials, including the pivotal trial of sepofarsen for Leber congenital amaurosis 10. ProQR is implementing mitigation procedures that support a rapid ramp up in enrollment as soon as the disruption resolves, including additional patient identification activities and documentation for additional site activations, while prioritizing the safety of trial participants and healthcare providers. For the trials of QR-421a and QR-1123, patients have already been identified for the next dose cohorts and the Company expects to begin dosing as soon as practical after clinical sites are ready and able to do so. This will be the same for the start of the clinical trial for QR-504a. The Company currently does not believe that its supply chain will be affected.

Due to the COVID-19-related delays, the Company has undertaken a budget review process and now anticipates its cash runway will fund operations into the second half of 2022.

“In these uncertain times, the health and safety of patients in our trials, their caregivers, and our employees remains our top priority. We believe we have taken appropriate measures designed to limit their risk,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “While we manage the challenges stemming from the COVID-19 pandemic, we remain confident in the fundamentals of our business, as demonstrated in part by the data we are sharing today from our QR-421a program. We have a productive platform, a deep pipeline of RNA therapies focused on inherited retinal diseases, and the benefit of a strong balance sheet, which positions us well to endure the current disruption and continue the important work with the communities we serve.”

Conference Call

Management will discuss the data and next steps for development during a webcasted conference call today, March 31, 2020, at 8:15 a.m. ET. The live and archived webcast of this presentation can be accessed through the Events and Presentations page on the Investors section of the Company’s website, www.ProQR.com. The dial-in details for the call are +1 631-510-7495 (US), +31 (0) 207143545 (NL), conference ID: 5986384. The archived webcasts will be available for approximately 30 days following the presentation date.

About Usher Syndrome Type 2 and Non-Syndromic Retinitis Pigmentosa

Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2 are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2 and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

About QR-421a

QR-421a is being evaluated in the Phase 1/2 Stellar trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and Rare Pediatric Disease designations from the FDA.

About the Stellar Phase 1/2 Trial of QR-421a

Stellar, or PQ-421a-001, is a first-in-human study of QR-421a in adults who have vision loss due to mutations in exon 13 of the USH2A gene and is conducted at expert sites in North America and Europe. It is a double-masked, randomized, 24-month study exploring the safety and efficacy of a single intravitreal injection of several dose levels of QR-421a and a control sham procedure into one eye.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof, our other programs and business operations, including timing of commencing clinical trials and enrollment of patients therein, the expected impact of the COVID-19 on our business operations, including our research and development plans and timelines and the supply chain for our clinical and development programs, and our financial position and cash runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; and general business, financial and accounting risks and litigation. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

Cautionary Note on Future Updates

The statements contained in this press release reflect our current views with respect to future events, which may change significantly as the global consequences of the COVID-19 pandemic rapidly develop. Accordingly, we do not undertake and specifically disclaim any obligation to update any forward-looking statements.

ProQR Therapeutics N.V.

PARIS--(BUSINESS WIRE)--SparingVision, a biotechnology company specializing in the research and development of innovative therapies for treating hereditary retinal degenerative diseases such as retinitis pigmentosa, has today announced that it has been awarded non-dilutive funding of €2.5 million, having submitted a winning proposal for the European EIC Accelerator (SME instrument Phase 2) program.

This funding will help speed up SparingVision's clinical and regulatory development. The company is currently producing the clinical batches needed to initiate the first clinical trials in Europe and the United States in 2020. The PHENOROD (NCT03975543) retrospective natural history study of the disease has already taken place and the results will be published soon. The PHENOROD 2 prospective study will begin in the Fall of 2019.

“We are delighted to have won this European EIC Accelerator program and would like to offer our sincere thanks to all bodies of the EU Horizon 2020 program. This funding reflects the innovative nature of the gene-independent approach of the gene therapy developed by SparingVision for the treatment of retinitis pigmentosa, rare hereditary retinal degeneration that leads to blindness. It will enable us to speed up our development and position the company as a new stakeholder in the treatment of retinitis pigmentosa, and meet an unmet public health need”, explains Florence Allouche, SparingVision President. “We are particularly pleased as this is the first time SparingVision has submitted a proposal for this highly selective European program, as we are currently preparing for new fundraising action. EU applications require specific expertise and experience, so we would like to thank retina specialists in several leading centers throughout Europe, Israel and the United States as well as the Retina International patient organization and the Foundation Fighting Blindness for their support, and the European support teams of Bpifrance and Efficient Innovation for their methodology, assistance and, in particular, for sharing our desire to succeed.”

EIC Accelerator (SME Instrument) is a European public program that funds risk innovation in disruptive small businesses that have significant growth potential and global ambitions. EIC Accelerator is part of Horizon 2020 – the EU's €80 billion funding program for Research and Innovation for 2014-2020. For this April 2019 session, the success rate is 4.44% at the European level. Out of 136 proposals submitted by French companies, only 4 have been selected, and this includes SparingVision, the only biotech company. According to Venture Radar, 25% of companies funded under EIC Accelerator are among the fastest-growing European firms (10%).

About SparingVision 
SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent drug candidate to treat retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects 40,000 people in France and nearly 2 million worldwide. SparingVision is a spin-off of the Paris Vision Institute. Bpifrance, Foundation Fighting Blindness (US) and Voir & Entendre Foundation invested €15.5 million in the company. SparingVision was laureate and Grand Prize of i-Lab 2017, the National Contest for the Creation of Innovative Companies, and is part of the first selection of companies of Hub Heath Tech launched by Bpifrance in December 2017. Florence Allouche, President of SparingVision, has won the Mercures Entrepreneurs Prize and the Women Trajectory’s award from HEC Paris and was elected "Woman of the Year 2017” by the financial magazine La Tribune.

www.sparingvision.com

Contacts

Florence Allouche 
Pharm D. President SparingVision, 
+33 1 43 46 20 60 
fag@sparingvision.com

Florence Portejoie 
FP2COM 
+ 33 6 07 76 82 83 
fportejoie@fp2com.fr

LEIDEN, Netherlands and CAMBRIDGE, Mass., March 11, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced the first patient dosed in the Phase 1/2 STELLAR clinical trial for QR-421a in patients with Usher syndrome type 2 or non-syndromic retinitis pigmentosa (RP). Interim data from the study are expected to be announced mid-2019.

"There are no effective treatments for most inherited retinal diseases, including Usher syndrome, and blindness often results,” said David G. Birch, Ph.D., Principal Investigator of STELLAR and ScientificDirector of the Retina Foundation of the Southwest in Dallas, Texas. “The STELLAR study is one of the first studies of its kind exploring the impact of ProQR’s RNA therapies on patients with Usher syndrome due to an Exon 13 mutation. The STELLAR trial will explore whether QR-421a (ProQR’s RNA therapy) can slow disease progression or even reverse it. Treatments such as this, that target the underlying cause of a disorder, have the potential to give new hope to patients and their families that life-changing therapies could be available in the near future.”

“Usher syndrome is a devastating disease, so we are pleased to advance QR-421a into the clinic with the goal to make a difference for these patients, similar to what we have observed with early but promising data for sepofarsen in patients with LCA10,” said Daniel A. de Boer, chief executive officer of ProQR. “We are committed to rapidly advancing our promising RNA therapies for inherited retinal diseases and we believe our platform of generating targeted RNA therapies with long retinal half-lives and the ability to reach both central and peripheral retina, we will be able to target many of these diseases in the coming years.”

Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the USH2A gene targeted by QR-421a cause vision loss in approximately 16,000 individuals in the Western world.

About the Phase 1/2 “STELLAR” trial

STELLAR, or PQ-421a-001, is a first-in-human study that will initially include approximately 18 adults who have vision loss due to mutations in exon 13 of the USH2A gene and will be conducted at about seven expert sites in North America and Europe. It will be a double-masked, randomized study exploring several dose levels and a control (sham injection), given as a single intravitreal injection of QR-421a into one eye. The first patient at each dose level will be dosed in an open-label manner. The objectives of the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by stopping progression or improvement of visual function and retinal structure through ophthalmic endpoints such as visual field, visual acuity and optical coherence tomography. Changes in quality of life in the trial subjects will also be evaluated.

Preliminary data from the first-in-human study are expected in mid-2019. Patients completing this trial will be able to participate in an extension study if eligible. Results from the single dose trial will inform the next stage that will potentially include a seamless adaptive, multi-dose, controlled trial with projected readout in 2021.

About QR-421a

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation from the FDA.

About Usher Syndrome

Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. Usher syndrome type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. To date, there are no approved treatments or products in clinical development that treat the vision loss associated with Usher syndrome type 2.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, Usher syndrome type 2 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. 
*Since 2012*

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-421a and its clinical development and therapeutic potential, including commencement of the STELLAR trial, trial design and timing of results from this trial. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, regulatory review or approval process, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:
Lisa Hayes
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855 
lhayes@proqr.com 

 

ProQR Therapeutics N.V. logo

 

Source: ProQR Therapeutics N.V.

Today, the Foundation Fighting Blindness announced the creation of the first-ever Retinal Degeneration Fund (RD Fund), a wholly owned, 501(c)(3) not-for-profit subsidiary focused on making venture philanthropy investments to further the Foundation’s mission: To provide preventions, treatments and cures for inherited retinal diseases and age-related macular degeneration as fast as possible. The RD Fund, which now has more than $70 million in initial funding, will invest in companies with projects nearing clinical testing.

“We are excited to initiate a focused and concentrated effort on our mission-related investments with the creation of the RD Fund,” said Benjamin Yerxa, PhD, CEO, RD Fund and Foundation Fighting Blindness. “With more than $70 million in assets to put to work, the RD Fund will support a portfolio of projects that advance our organization’s mission.”

David Brint, chairman of Foundation Fighting Blindness added, “We are adapting to a rapidly changing environment, and the RD Fund is part of our strategy. Translational R&D is increasing in cost, and our supporters want to see a disciplined and professional approach to maximally leverage these hard-earned funds to provide clear, measurable progress, as well as the potential for a sustainable financial future. It is imperative that the most promising research has the best opportunity to reach patients in need.”

The new RD Fund is also the vehicle for Foundation Fighting Blindness’ recent mission-related investments. The Foundation made its first such investment in SparingVision in 2016, through the Foundation’s subsidiary, the Clinical Research Institute (CRI). Since that time, through CRI, the Foundation has co-funded projects with its partners, Nacuity Pharmaceuticals and ProQR. Recognizing the increasing need for more investment to speed translation of scientific discoveries into meaningful clinical results, Foundation Fighting Blindness is transforming its CRI subsidiary into the RD Fund, which will now focus purely on mission-related investments.

Clinical research activities formerly overseen by CRI will now be managed by a new Foundation Fighting Blindness subcommittee chaired by Dr. Frederick Ferris III, former clinical director of the National Eye Institute.

“We would like to thank the Foundation’s Clinical Research Institute board members for their many years of service and dedication to the success of the institute,” said Dr. Morton Goldberg, chairman of the former CRI, who will continue his service on the Foundation’s Research Oversight Committee. “The ability to focus separately on clinical research, now under Rick 
Ferris’ leadership, and to manage the translational investments through the RD Fund are going to be powerful forces moving the field forward.”

An independent board of directors will oversee the RD Fund, with representatives from Foundation Fighting Blindness and several at-large members who bring significant outside expertise to the team. Warren Thaler, Chairman of the RD Fund Board, said, “I’m excited to use my investment experience to further the foundation’s mission, and am humbled to work with a world-class group of directors to oversee the RD Fund.” In addition to Mr. Thaler, the initial board members include: David Brint, Eugene de Juan, MD, Jacque Duncan, MD, Adrienne Graves, PhD, Kelly Lisbakken, Jason Morris, and Jonathan Steinberg, MD.

The RD Fund will be managed by Benjamin Yerxa, PhD, CEO, Jason Menzo, COO, and Russell Kelley, PhD, MBA, vice president of investments and alliances, along with additional support from the Foundation staff and outside consultants. The $70 million of initial assets include the first three investments made by the former CRI. The RD Fund will remain open to new donations for several months. All proceeds from investments will be used to fund the Foundation’s research mission. In parallel, the Foundation is dedicated to providing continued, robust funding of the best academic laboratories in the inherited retinal disease field to provide a pipeline of future therapies.

“Although $70 million sounds like a lot of money, we have to remember that a single R&D program can cost tens of millions of dollars or more,” said Paul Manning, whose family recently completed a Gund Challenge pledge of nearly $12 million directed to the RD Fund. “The Manning family is truly excited to see the Foundation Fighting Blindness move in this direction, and we invite others to join us in financially supporting this effort.”

ABOUT THE RETINAL DEGENERATION FUND
The Retinal Degeneration Fund (RD Fund) is a 501(c)(3) not-for-profit subsidiary of the Foundation Fighting Blindness. Launched in 2018 with a $70 million initial investment, the RD Fund focuses on making venture philanthropy investments in companies with projects nearing clinical testing.

ABOUT FOUNDATION FIGHTING BLINDNESS
The Foundation Fighting Blindness was established in 1971. It has since raised more than $750 million for research aimed at preventing, treating and curing blindness caused by retinal degenerative diseases. In excess of 10 million Americans, and millions more worldwide, have experienced or are at risk of experiencing vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to improve the lives of people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases. Learn more athttp://www.blindness.org/.

For more information about the RD Fund and major gift contributions, please contact rustykelley@fightblindness.org.

  • Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
  • Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic.
  • QR-421a received orphan drug designation from the FDA.
  • There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A.
  • QR-421a is part of ProQR’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QR-411 that addresses another genetic mutation resulting in Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy. 
  • QR-421a is expected to advance towards the clinic in 2018, with clinical data expected in 2019.

COLUMBIA, Md. and LEIDEN, the Netherlands, Feb. 12, 2018 (GLOBE NEWSWIRE) -- Foundation Fighting Blindness and ProQR Therapeutics N.V. (NASDAQ:PRQR), today announced that they have entered into a partnership to develop QR-421a for Usher syndrome 2A caused by an exon 13 mutation of the causative USH2A gene.  Under the agreement, Foundation Fighting Blindness will provide up to $7.5 million in funding to ProQR for the preclinical and clinical development of QR-421a, which is expected to advance towards the clinic in 2018, and safety and efficacy results from the Phase 1/2 trial in Usher syndrome patients are expected in 2019.

Usher syndrome is a devastating genetic disease in which patients first develop hearing loss and then progressive vision loss, thereby threatening their independence and quality of life. Currently there is no treatment for the ophthalmic manifestation of Usher syndrome type 2A.  QR-421a is a first-in-class RNA oligonucleotide that is being developed for the treatment of vision loss associated with the disease. QR-421a is designed to modify the RNA such that functional usherin protein is produced in the retina with the goal of stopping the progression of the disease and potentially gaining peripheral vision. ProQR in-licensed the technology underlying QR-421a from Radboud University Medical Center in the Netherlands, where it was invented by lead investigator Dr. Erwin van Wyck.

Foundation Fighting Blindness’ Clinical Research Institute (FFB-CRI) has also launched a natural history study in 120 people with USH2A mutations. The study — known as RUSH2A (“R” stands for “rate of progression”) — was launched in 2017 and is being conducted at about 20 clinical sites around the world. RUSH2A investigators will use a variety of technologies to monitor changes in vision and retinal structure to document and analyze disease progression. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.

“Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness.  “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.”

QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials.  The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and pediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene.  This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE-40 mutation, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.

“We are excited to team up with the Foundation Fighting Blindness to develop QR-421a for patients that suffer from Usher syndrome due to exon 13 mutations," said Daniel A. de Boer, CEO of ProQR. “They are the leading private funder of retinal disease research with a very patient centric approach which is a core pillar of our strategy. Through this partnership with the Foundation we plan to gain access to important know-how to develop programs in retinal diseases. We expect that the additional funding will allow us to rapidly advance this novel therapy for this orphan disease with a severe unmet need.”

About Foundation Fighting Blindness

The Foundation Fighting Blindness was established in 1971 and has raised more than $725 million for research on preventing, treating and curing blindness caused by inherited retinal diseases. In excess of 10 million Americans, and millions more worldwide, experience or are at risk for vision loss due to retinal degenerations. Through its support of focused and innovative science, and by teaming with industry, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

About QR-421a

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome 2A due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to repair the genetic defect in the RNA in the eye, such that it leads to the expression of a shortened but functional protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation from the U.S. Food and Drug Administration. 

About Usher Syndrome Type 2A

Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. Usher syndrome type 2A is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with Usher syndrome type 2A.

PROQR FORWARD-LOOKING STATEMENTS 

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding our collaboration with Foundation Fighting Blindness, including statements regarding the expected funding and benefits of such collaboration, our product candidates QR-110, QRX-1011, QRX-504 and QR-421a, including their clinical development and therapeutic potential, including future development plans.  Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, and that we may not be able to realize the potential benefits of orphan drug exclusivity, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.:

Investor and Media Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com

 

Sources: ProQR Therapeutics and Foundation Fighting Blindness

Primary Logo

 

Source: ProQR Therapeutics N.V.

The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has announced an investment of up to $7.5 million to advance a promising, emerging drug treatment for retinitis pigmentosa (RP) into and through a Phase II clinical trial. Known as N-acetylcysteine-amide (NACA), the molecule is designed to slow vision loss by protecting retinal cells from oxidative stress. Oxidative stress is a process that accelerates and exacerbates degeneration in many inherited retinal conditions. In several in vitro and in vivo models, including previous FFB-funded lab studies of rodent models at Johns Hopkins University, NACA slowed retinal degeneration.

Nacuity Pharmaceuticals, Inc., a start-up company in Fort Worth, Texas, owns the rights to NACA for ophthalmology and will be developing the drug with support from FFB-CRI. As part of its agreement with Nacuity, upon commercialization of NACA, FFB-CRI will be entitled to royalty payments from future NACA sales.

“Based on the knowledge we’ve gained in lab studies, we are excited about NACA’s potential for saving vision,” said Patricia Zilliox, Ph.D., chief drug development officer at FFB-CRI. “Oxidative stress causes cell degeneration and vision loss in virtually all forms of RP, so we are hopeful that NACA, with its anti-oxidative properties, can benefit most people with RP, regardless of the gene mutation causing their disease.”

NACA is derived from N-acetylcysteine (NAC), a drug approved by the U.S. Food & Drug Administration (FDA) for the treatment of acetaminophen overdose by lessening hepatotoxicity. The benefits of NACA over NAC are believed to include greater lipophilicity and tissue penetration therefore increasing anti-oxidative properties in the retina.

“We are excited to make a significant investment in NACA, because the drug has an opportunity to help a substantial number of people with RP, a blinding retinal disease affecting 100,000 people in the U.S., and more than 2 million worldwide,” said William T. Schmidt, chief executive officer of FFB. “The fact that NACA is derived from an FDA-approved drug also boosts our confidence that it can make it through the clinical development process and out to the people who need it.”

“We are pleased and excited to have the support of FFB in our quest for an effective treatment for patients with RP.” said Halden Conner, President of Nacuity. “The review of, and input to, our plans by their independent, renowned ophthalmological experts will markedly increase our odds of success in bringing this treatment to the RP community.”

“Drug substance process manufacturing, drug product development and toxicology studies are planned for 2017 to facilitate an investigational new drug application (IND) and initiation of a Phase I Clinical Safety Program for this new chemical entity,” Nacuity’s Vice-President of Research and Development, G. Michael Wall, Ph.D., stated.  Nacuity hopes to launch a Phase II clinical trial for NACA in early 2018.

The Foundation Fighting Blindness was established in 1971. It has since raised more than $700 million for research aimed at preventing, treating and curing blindness caused by retinal degenerative diseases. In excess of 10 million Americans, and millions more worldwide, have vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases. 

Nacuity Pharmaceuticals, Inc. was formed in May 2016 to focus on bringing a potential treatment with NACA to patients with RP guided by the groundbreaking work by Peter Campochiaro, MD, at the Wilmer Institute of Johns Hopkins University.

The development of a vision-saving treatment for people with retinitis pigmentosa (RP) is getting a major boost thanks to the formation of the French biotech SparingVision to move it into a clinical trial and out to the international marketplace.

A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. SparingVision’s goal is to launch a clinical trial for the protein in 2019.

The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI), Bpifrance — Biotechnology Acceleration Fund and Innovative Biotherapies and Rare Diseases Funds created by the French national program (BPI), and the Fondation Voir et Entendre (FVE) have announced a total of €15.5 million in tranche funding for the development of RdCVF. FFB-CRI and BPI are each investing €7 million with FVE providing €1.5 million.

SparingVision also received a €300,000 award known as the Honor Prize from the French Ministry of Research. The award is given to new, innovative companies in France competing in a national contest.

RdCVF is a naturally occurring protein in the retina identified by SparingVision co-founders José Sahel, MD, and Thierry Léveillard, PhD, at the Institut de la Vision. The scientists demonstrated in laboratory studies that RdCVF prevented or slowed the degeneration of cones, the cells in the retina that provide central and color vision and enable people to read, drive, and recognize faces. RdCVF is naturally secreted by rods, the retinal cells that provide night and peripheral vision.

RP is a genetic condition affecting about two million people worldwide. The retinal disease is usually diagnosed in childhood, progressively leading to legal or total blindness in adulthood. RP initially affects rods. The progressive loss of rods leads to loss of cones. There are currently no therapies for RP.

“Saving retinal cone cells is critical for preserving vision for people with genetic retinal diseases,” says Dr. Sahel.

“After several years of investigation, we understand the mechanism of action for RdCVF and have demonstrated its strong efficacy in several lab studies,” says Dr. Léveillard.

“SparingVision’s emerging therapy has the potential to save the vision of millions. I am delighted by our partners’ investment to get RdCVF out to the people who desperately need it,” says Florence Allouche Ghrenassia, PharmD, president at SparingVision. She brings 16 years of experience at Assistance Publique Hôopitaux de Paris as TTO Director in advancing innovative early stage therapies into the clinic to her new role.

FFB-CRI provided much of the earlier research funding to develop RdCVF. “We have been excited about this therapy’s potential for saving vision and therefore committed significant resources to boost its development,” says Patricia Zilliox, PhD, FFB-CRI’s chief drug development officer. “The establishment of SparingVision and the investment by our partners are essential to getting the treatment into the marketplace. We are pleased to be a part of this translational process.”