March 02, 2026-
Opus Genetics has announced early findings from its ongoing Phase I/II clinical trial evaluating OPGx-BEST1, an investigational AAV gene therapy for patients with best vitelliform macular dystrophy (BVMD) and autosomal-recessive bestrophinopathy (ARB). The data were presented at the Macula Society annual meeting in San Diego.
The update included results from a 63-year-old female participant with ARB treated with a single subretinal injection of OPGx-BEST1. At three months post-treatment, the AAV gene therapy was reported to be well tolerated, with no ocular inflammation, serious adverse events, or dose-limiting toxicities observed.
Encouraging early signals of biological activity were also reported. The treated eye demonstrated a 12-letter gain in best corrected visual acuity (BCVA). Central subfield thickness decreased by 23%, and intraretinal fluid resolved within one month in retinal areas with limited atrophy. These findings suggest potential functional and anatomical improvements following AAV-mediated BEST1 gene delivery.
The open-label, adaptive Phase I/II study is evaluating a one-time subretinal administration of OPGx-BEST1 in one eye per participant across two adult dosing cohorts. The primary objectives are to assess safety and tolerability, identify an optimal dose for future development, and monitor longer-term outcomes through functional and structural retinal endpoints.
According to CEO George Magrath, the early three-month data from the sentinel participant represent an important milestone for the OPGx-BEST1 program and for individuals living with BEST1-related inherited retinal diseases.
Recruitment is ongoing at two U.S. clinical sites, with expansion planned to additional centers in Cincinnati, Florida, and New York. Two participants are currently enrolled, and three-month data from the full Cohort 1 are anticipated in mid-2026.
