The emerging therapy improved vision for some participants in an earlier clinical trial.

Durable vision improvements were also observed out to 18 months for previously dosed adult patients in the trial.

Lance Baldo, MD, CEO at Beacon Therapeutics, talks to host Ben Shaberman about his company's fully enrolled Phase 3 VISTA clinical trial for laru-zova, a gene therapy for X-linked retinitis pigmentosa (RPGR mutations) that's showed promising results in earlier trials. They also discuss Beacon's forthcoming clinical trial for its dry age-related macular degeneration gene therapy.

The documentary “Aggie: An Incredible Story of Art and Justice” chronicles her story.

NACA is a gene-agnostic, oral medication designed to preserve vision in people with retinitis pigmentosa and Usher syndrome.

September 5, 2025. Dr. George Magrath, CEO of Opus Genetics, talks with host Ben Shaberman about the company's evolution and progress in clinically developing gene therapies for several inherited retinal diseases including those caused by mutations in the genes: LCA5, BEST1, RHO, RDH12, MERTK, NMNAT1, and CNGB1.

The Foundation funded earlier, proof-of-concept studies for the Best1 gene therapy and later launched Opus to develop several IRD gene therapies.

The trial is evaluating a cell dose that is 50 percent higher than the highest dose used in a previous Phase 2b trial.

August 8, 2025. Dr. Christina Ohnsman talks to host Ben Shaberman about Tern Therapeutics, a start-up company she co-founded to develop gene therapies for Batten disease and potentially other conditions. Dr. Ohnsman reviews early results for a Batten disease (CLN2) retinal gene therapy clinical trial. She also reflects on her previous work as a pediatric ophthalmologist and roles with Spark Therapeutics and Regenxbio.

Susvimo is an FDA-approved treatment that continuously delivers drug through a tiny, implanted capsule