Roche digs deeper into gene therapy for the eye
Roche is putting more money into gene therapies for eye diseases, announcing a deal to work with University of Pittsburgh spinout Avista Therapeutics to develop better delivery tools for the complex treatments.
Through the deal, Avista will develop new capsids, or viral shells capable of delivering genetic material into cells, that fit a “profile defined by Roche,” the companies said in a statement. Avista will get $7.5 million upfront in the alliance, but could receive up to $1 billion if various milestones are met and additional fees are collected.
The deal suggests Roche is diving even more deeply into gene therapy, and in particular, gene-based treatments for eye diseases. Through its nearly $5 billion buyout of Spark Therapeutics in 2019, Roche gained the gene therapy approved by U.S. regulators, Luxturna, which treats an inherited form of blindness. That deal also handed Roche two experimental hemophilia treatments, as well as other programs in earlier stages of development.
Eye diseases are particularly attractive for gene therapy developers because the organ is protected from certain immune responses that can neutralize foreign invaders, like the adeno-associated viruses, or AAVs, often used to deliver treatments. The promise of treating eye diseases with gene therapy has attracted the interest of big drugmakers like Novartis, Biogen and Johnson & Johnson. Smaller companies like Regenxbio and Adverum Biotechnologies are testing gene therapies against common forms of vision loss, too.
Under the agreement signed Tuesday, Avista will make capsids and give Roche the right to license them, after which the Swiss drugmaker will be responsible for developing the gene therapies that come from the work. Those Roche-licensed projects will be distinct from Avista’s internal pipeline.
The deal comes amidst a retrenchment in the gene therapy field, as several companies have restructured or cut jobs in recent months. Clinical, regulatory and safety setbacks have each chilled investor interest, and stock prices have tumbled during a sector-wide downturn.
Avista, meanwhile, was spun out by the University of Pittsburgh in October 2021 and seeded with $10 million by the institution’s technology transfer arm, UPMC Enterprises. The company was founded by Leah Byrne, an assistant ophthalmology professor of University of Pittsburgh medical school, who serves as chief scientific officer; José-Alain Sahel, the University of Pittsburgh’s ophthalmology chair; and former National Eye Institute director Paul Sieving.
The startup is built around gene therapy technology that’s meant to target diseased eye cells better than existing methods.
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