Opus reports early vision improvements in BEST1 and LCA5 gene therapy studies
Published in Diseases!, Drugs development & discovery.
Published October 16, 2026.
Opus Genetics reported early clinical findings from two investigational retinal gene therapies on October 15 at Retina in Rio. The programs target inherited vision disorders associated with BEST1 and LCA5 mutations.
For OPGx-BEST1, the company reported improved retinal sensitivity in three of four evaluable participants in the initial low-dose BIRD-1 cohort. This small denominator matters: the reported 75% figure is not an established response rate for the broader patient population.
For OPGx-LCA5, improvements in visual acuity and light sensitivity were observed in three adults through 24 months and three children through six months. The company expects dosing in its Phase 3 study to begin in the fourth quarter of 2026; that remains a planned milestone.
Evidence limits: These are sponsor-reported findings from small early-stage cohorts. They do not establish a cure, comparative effectiveness or the full safety profile. Larger studies and longer follow-up are needed to assess how reliably benefits persist and which patients may respond.