Gene therapy is giving people born with blinding diseases the sight of their families back

At the Retina in Rio 2026 conference, Opus Genetics shared early results from two gene-therapy trials for inherited retinal diseases that blind people from birth or childhood. In the BEST1 trial, three of four evaluable participants in the low-dose group gained retinal sensitivity within months. In the LCA5 trial, six participants — three adults and three adolescents — kept their vision gains for up to two years, with some measures of cone vision (the sharp, daylight kind) restored toward normal ranges; participants described recognising family members and getting around on their own again. The data are early and come from the company, and a larger Phase 3 trial is expected to start dosing this year — but for families who expected only darkness, the direction is unmistakable.

SDG 3 · Good health and well-being — Mechanism: tech innovation