Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027
Opus Genetics announced positive low-dose Cohort 1 data from its Phase 1/2 trial of OPGx-BEST1, showing a favorable safety and tolerability profile with no serious adverse events and clinically meaningful improvements in visual function for all five participants. The company also completed a successful FDA Type C meeting aligning on a potential pivotal endpoint, with potential Phase 3 dosing in 2027 and cash runway into 2029.
Why it matters — The encouraging early safety and efficacy data, plus FDA alignment on a pivotal endpoint, could advance this gene therapy candidate toward later-stage trials for BEST1-related inherited retinal disease.