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Opus Genetics Reports Phase I/II Trial Results of OPGx-BEST1 Gene Therapy

Opus Genetics presented early Phase I/II data for its OPGx‑BEST1 gene therapy targeting best vitelliform macular dystrophy and autosomal‑recessive bestrophinopathy. In a sentinel 63‑year‑old participant, the treatment was well tolerated and delivered a 12‑letter gain in best‑corrected visual acuity after three months, alongside a 23% reduction in central subfield thickness and rapid resolution of intraretinal fluid. The open‑label study administers a single subretinal injection per eye across two dosing cohorts, with two participants currently enrolled and full Cohort 1 results slated for mid‑2026. Recruitment is expanding to additional U.S. sites.