Opus Genetics posts OPGx-BEST1 Phase 1/2 low-dose cohort presentation update
IRD•Phase 1/2 low-dose cohort update for OPGx-BEST1
Opus Genetics published 3-month Phase 1/2 results for OPGx-BEST1 gene therapy in low-dose Cohort 1 for BEST1-related retinal disease.
- Safety profile: no serious adverse events, no dose-limiting toxicities, no intraocular inflammation in 5 treated participants.
- Early efficacy signals: 75% of evaluable participants met an FDA-aligned microperimetry threshold of at least 3 dB improvement in 5 loci.
- Manufacturing timeline: Phase 3 and commercial supply targeted for early 2027; pivotal trial dosing planned in 2027.
