Opus Genetics posts OPGx-BEST1 Phase 1/2 low-dose cohort presentation

• Opus Genetics released 3-month results from the low-dose Cohort 1 of its Phase 1/2 OPGx-BEST1 gene therapy study.
• Low-dose cohort reported no serious adverse events, no dose-limiting toxicities, no intraocular inflammation across 5 treated participants.
• Microperimetry endpoint aligned with FDA: ≥3 dB improvement in ≥5 prespecified loci; 75% of evaluable participants met threshold.
• Structural improvements reported in 80% of participants at 3 months, led by reduced vitelliform material in BVMD and reduced intraretinal fluid in ARB.
• Pivotal trial expected to start dosing in 2027; Phase 3 and commercial manufacturing targeted for early 2027 delivery.

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