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
July 25, 2025. Dr. Christine Curcio, one of the world's foremost experts in age-related macular degeneration (AMD) pathogenesis and the associated risk of advanced vision loss, talks to host Ben Shaberman about her epical career and recent Proctor Medal lecture with Dr. Cynthia Owsley at the 2025 meeting of the Association for Eye and Vision Research (ARVO). Her work has led to a highly effective test for predicting who is at greatest risk of losing vision from AMD.
The therapy delivers copies of the RORA gene to address multiple disease pathways.
The company plans to complete BLA submission in early 2026.
July 11, 2025. Richard Small, CEO of Neurotech, talks to host Ben Shaberman about ENCELTO™, the first and only FDA-approved treatment for adults with idiopathic Macular Telangiectasia Type 2 (MacTel). The therapy is a tiny, implantable capsule, the size of a rice grain, that delivers neurotrophic factors to preserve the patient's retinal cells and vision. The approach was previously developed to treat retinitis pigmentosa and age-related macular degeneration.
The expanded Phase 1/2/3 shortens time to potential FDA approval.
June 13, 2025. Dr. Philip Beales, a co-founder of Axovia Therapeutics, talks to host Ben Shaberman about Bardet Biedl syndrome, ciliopathies, and his company's emerging gene therapy for Bardet Biedl syndrome type 1 (BBS1) which is moving toward a clinical trial.
May 23, 2025. Lenore von Krusenstiern, MD, PhD, at BlueRock Therapeutics talks to host Ben Shaberman about the company's clinical trial for a photoreceptor replacement therapy for people with inherited retinal conditions such as retinitis pigmentosa and cone-rod dystrophy. The approach, which uses induced pluripotent stem cells to develop photoreceptor precursors, comes out of the lab of stem cell pioneer David Gamm, MD, PhD, University of Wisconsin-Madison.
Despite missing the primary endpoint, the treatment improved vision for some patients.
The company is planning a Phase 2/3 clinical trial for therapy in late 2025.
Part B of the trial will enroll nine adult patients as well as three pediatric patients.