• 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.

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