PRPH2 Workshop 2026: Day 1 Keynote – John Samuel

Dr. Steven Johnston, co-founder of the non-profit organization A Race Against Blindness, talks about his son’s diagnosis with Bardet-Biedl syndrome – a rare genetic condition that causes progressive vision loss, his partnership with the Foundation Fighting Blindness and how his foundation turns luxury car giveaways into funding for research on potential treatments.

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 gene, according to a press release from Opus.

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 less advanced disease.

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 clinical trial. Beacon plans to begin seeking FDA approval later this year. If approved, this would be the first treatment available for XLRP, one of the more severe forms of retinitis pigmentosa that predominantly affects males.

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 vision in people with severe vision loss from RP.

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.