Opus Genetics reported new early clinical data for its OPGx-BEST1 gene therapy in patients with BEST1-related inherited retinal diseases (IRDs). The results, presented at the 49th annual meeting of the Macula Society in Coronado, California, covered the first adult participant in the ongoing phase 1/2 study.

The sentinel patient, a 63-year-old female with autosomal-recessive bestrophinopathy (ARB), showed favorable safety and tolerability 3 months after a single subretinal injection. No ocular or treatment-related adverse events were observed, and no dose-limiting toxicities were reported. Functional improvements included a 12-letter gain in best-corrected visual acuity (BCVA) in the treated eye, while structural measures showed a 23% reduction in central subfield thickness and resolution of intraretinal fluid in areas with less atrophy. Investigator Mark Pennesi, MD, PhD, noted that the results support continued evaluation of gene augmentation for BEST1-associated disease, which currently has no approved treatments.

The phase 1/2 study is currently recruiting at a pair of US sites, with additional locations planned in Florida, Ohio, and New York. Full 3-month results for the first cohort are expected in mid-2026. The trial aims to determine safety, tolerability, and biological activity, including visual and anatomical outcomes. RP