Foundation Fighting Blindness

An emerging gene therapy treatment for Leber congenital amaurosis (LCA) received the green light from the U.S. Food and Drug Administration to proceed with clinical trials in people, bringing it one step closer to patients with a rare form of childhood blindness. The potential treatment from Opus Genetics – OPGx-RDH12 – is targeted to patients who have LCA associated with mutations in the RDH12 ge...

Host Maddie Mossman talks with Dr. Daniel Chung, Chief Medical Officer of Beacon Therapeutics, about Laru-zova, an emerging treatment for X-linked retinitis pigmentosa. Based on encouraging clinical trial results, Beacon plans to begin a rolling FDA submission for the potential gene therapy later this year. The Foundation funded pre-clinical research that went on to became laru-zova.

PRPH2 Workshop 2026: Day 1 Keynote – John Samuel by Foundation Fighting Blindness

Opus Genetics has shared encouraging early results from its ongoing Phase 1/2 clinical trial for OPGx-BEST1, a potential one-time gene therapy for people with BEST1-related retinal disease. All five patients enrolled in the clinical trial had clinically meaningful improvement in visual function, according to Opus Genetics. The greatest functional improvements were observed in participants with les...

Beacon Therapeutics reported positive results from the Phase 3 pivotal trial of laru-zova, its potential gene therapy treatment for X-linked retinitis pigmentosa. A significant proportion of patients improved dim-light vision by 15 or more letters after treatment with either high or low doses of laru-zova compared to the group that was not treated, according to preliminary results from the VISTA c...

The U.S. Food and Drug Administration accepted Dallas-based Nanoscope Therapeutics' biologics license application for MOGENRY, an emerging treatment for adults with retinitis pigmentosa and severe vision loss. The investigational treatment does not require a genetic diagnosis or specific gene mutation, giving MOGENRY the potential to become the first approved gene-agnostic treatment to improve vis...

Two non-profit professionals talk about plugging into community and finding low-vision resources through the Foundation

Sumitomo Pharma America treated its first patient in a Phase 1/2a clinical trial for an emerging retinitis pigmentosa (RP) treatment called DSP-3077. The clinical trial plans to treat 12 adults with one of two different doses to evaluate safety and effectiveness of the treatment among patients who have RP.

Tarsus Pharmaceuticals announced plans to acquire Alkeus Pharmaceuticals, including its emerging oral treatment for Stargardt disease, gildeuretinol (ALK-001), in a deal valued up to $800 million. Stargardt disease is the most common form of inherited macular degeneration and is often diagnosed in children or teens. There are no current treatments.

In Europe, Science Corporation has commercially launched PRIMA, a retinal implant designed to restore central vision in people with geographic atrophy (GA). The treatment has been shown in clinical trials to restore functional central vision in people with GA who until now had no way to get lost central vision back. While PRIMA is available in Europe, it is still going through FDA approvals in the...

Belite Bio’s tinlarebant could become the first-ever approved treatment for Stargardt disease

Dr. Alicia Kemble explains how the Foundation is using venture philanthropy to drive treatments for inherited retinal diseases even closer to the finish line.

Sepul Bio recently announced that it completed enrollment of its Phase 2b safety and efficacy clinical trial for ultevursen – an investigational RNA therapy to treat patients who have retinitis pigmentosa (RP) associated with mutations in the USH2A gene.

FDA approves Lumvoa, a new treatment for thyroid eye disease by Foundation Fighting Blindness

Belite Bio submits new drug application to FDA for its Stargardt treatment by Foundation Fighting Blindness

New host Maddie Mossman interviews retinal surgeon Dr. Staropoli, the first medical doctor to win a NASCAR race.

Promising oral treatment moves closer for Stargardt patients.

2020 Llura Liggett Gund Award recipient honored for pioneering gene therapy that transformed vision research

Data monitoring committee recommends proceeding with registration-enabling study for ATSN-201

MeiraGTx Announces the Acquisition of Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-linked Retinitis Pigmentosa (XLRP) by Foundation Fighting Blindness

Strategic funding extends cash runway through 2029 and advances multiple inherited retinal disease programs

VG801 shows sustained vision improvements through 12 months with favorable safety profile

Potential cell therapy, DSP-3077, gains special regulatory status to support development for inherited retinal disease.

First-of-its-kind small molecule therapy targets protein misfolding at the root of RHO-adRP

First patient shows vision improvements with a good safety profile.