Clinical TrialMay 7, 2026, 04:32 PM
Opus Genetics' LCA5 gene therapy restores cone vision in pediatric patients
AI Summary
Opus Genetics announced positive clinical and preclinical data from its gene therapy programs at the ARVO Annual Meeting 2026. The OPGx-LCA5 program demonstrated robust restoration of cone-mediated vision in pediatric patients with severe LCA5, showing over 30-fold improvements in cone sensitivity and improved visual acuity. Early data from the OPGx-BEST1 program also showed visual acuity gains and improved retinal structure in adult patients. Additionally, preclinical RHO programs exhibited durable retinal preservation and supported clinical translation, highlighting a mutation-independent AAV approach for diverse retinal diseases.
Key Highlights
- OPGx-LCA5 showed over 30-fold improvements in cone sensitivity in pediatric LCA5 patients.
- OPGx-LCA5 demonstrated improved visual acuity and functional vision in treated eyes.
- OPGx-BEST1 showed up to 12-letter improvement in visual acuity in treated adult patients.
- OPGx-BEST1 resulted in ~23% reduction in central subfield thickness.
- Preclinical RHO programs demonstrated durable retinal preservation and structural rescue.
- OPGx-BEST1 potency assays showed consistent potency across manufacturing lots (50–150%).
- Opus Genetics expects 3-month topline data for OPGx-BEST1 Cohort 1 in September 2026.