September, 22 2021 • Articles

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

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


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.”