Luxturna inventor's research helps seal gene therapy biotech Opus Genetics' $19M debut

She planted a seed that led to vision loss gene therapy Luxturna, and now Jean Bennett, M.D., Ph.D., is behind another retinal gene therapy biotech. Opus Genetics emerged Wednesday with its own seed in the form of $19 million in funding. 

The seed financing will help Opus take Bennett's preclinical research at the University of Pennsylvania's School of Medicine, where she's an emeritus professor, into the clinic next year. The Foundation Fighting Blindness founded Opus through its Retinal Degeneration Fund.

RELATED: Roche bulks up in gene therapy and hemophilia with $4.3B Spark buyout

Among the first gene therapies approved for a genetic disease, Luxturna secured the FDA nod for an inherited form of vision loss in December 2017. The $850,000 drug was a key part of Spark's $4.3 billion exit to Roche in February 2019. Luxturna is also sold through partner Novartis in regions outside the U.S.

Roche does not currently break out Luxturna sales, but the therapy was estimated to be worth $326 million at the end of December 2020. 

Now, Bennett's research is being licensed by Opus for two programs aimed at addressing mutations in genes that cause Leber congenital amaurosis, or LCA, which comprises rare inherited retinal diseases present in infancy.

First up is a treatment targeting mutations in the LCA5 gene, which is behind one of the most severe forms of LCA. That asset is slated for clinical entry in the middle of 2022, the company said. The second program is focused on restoring protein expression to stop functional deterioration in patients with retinal dystrophy. 

RELATED: Novartis doesn't think optogenetics will completely cure blindness—but its new biotech is going to give it a try

Opus is being led by Foundation Fighting Blindness CEO Ben Yerxa, Ph.D., as acting CEO. 

Bennett's research is aided by Opus' other scientific founders, including Junwei Sun, chief administrator for Penn's Center for Advanced Retinal Ocular Therapeutics and Eric Pierce, M.D., Ph.D., an ophthalmology professor at Harvard Medical School.