Opus Genetics announced positive clinical data from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at the Retina in Rio 2026 conference. The results support the continued development of both therapies for inherited retinal diseases, and the company confirmed it expects to begin dosing for its OPGx-LCA5 Phase 3 trial in Q4 2026.
Key Details
- OPGx-BEST1 (Phase 1/2): Data from the initial low-dose cohort showed improved retinal sensitivity in 75% of evaluable participants (3 of 4), supporting further clinical development.
- OPGx-LCA5 (Phase 1/2/3): The therapy demonstrated sustained improvements in visual acuity and light sensitivity in adult participants through 24 months and encouraging results in pediatric participants through six months.
- Upcoming Milestone: The company expects to initiate dosing for the OPGx-LCA5 Phase 3 clinical trial in the fourth quarter of 2026.