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