Opus Genetics Reports Early Progress for BEST1 Gene Therapy

Opus Genetics has reported early findings from its ongoing Phase I/II trial of OPGx-BEST1, a gene therapy being developed for Best Vitelliform Macular Dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB). The results were presented at the Macula Society Annual Meeting in San Diego.

Initial data from a 63-year-old participant with ARB showed the therapy was well tolerated after three months, with no ocular inflammation, adverse events or dose-limiting toxicities reported.

Researchers also observed early functional improvement, including a 12-letter gain in best-corrected visual acuity (BCVA) in the treated eye. Central subfield thickness decreased by 23%, and intraretinal fluid resolved within one month in areas with limited atrophy.

The open-label study evaluates a single subretinal injection of OPGx-BEST1 in one eye across two dosing cohorts. Recruitment is ongoing at US sites, and full Cohort 1 results are expected in mid-2026.

10-03-2026