Opus Genetics announced positive interim data from Cohort 1 of its Phase 1/2 BIRD-1 clinical trial for OPGx-BEST1, a gene therapy for BEST1-related retinal diseases. All five participants treated at 1.5 x 10⁹ vg/eye demonstrated clinically meaningful improvements in visual function and structural metrics, with no serious adverse events reported. The company advanced to higher-dose Cohort 2 (4.5 x 10⁹ vg/eye), which is over-enrolled with eight participants; dosing completion is expected in Q4 2026. Additionally, Opus Genetics aligned with the FDA on potential pivotal trial endpoints and manufacturing requirements, targeting Phase 3 participant dosing to begin in 2027. The company operates as a clinical-stage biopharmaceutical firm developing gene therapies for inherited retinal diseases.
