Core Insights - Opus Genetics has initiated the dosing of the first participant in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease, marking a significant milestone for the inherited retinal disease community [2][5] - The OPGx-BEST1 therapy is a one-time subretinal injection aimed at restoring function to retinal pigment epithelium cells affected by mutations in the BEST1 gene [3][6] - Initial data from the trial is expected in Q1 2026, which will evaluate the safety, tolerability, and preliminary efficacy of the treatment [7] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases [10] - The company’s pipeline includes seven AAV-based programs, with OPGx-BEST1 targeting BEST1-related retinal degeneration [10] - The company is based in Research Triangle Park, NC, and is also advancing other therapies for various ocular conditions [10] Clinical Trial Details - The Phase 1/2 trial, known as BIRD-1, is an adaptive, open-label, dose-exploring study evaluating OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy or Autosomal-Recessive Bestrophinopathy [7] - The trial will explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure [7] - The study is being conducted by a team of experts from the Retina Foundation of the Southwest and Retina Consultants of Texas [4]
Opus Genetics Announces Dosing of First Participant in OPGx-BEST1 Phase 1/2 Gene Therapy Clinical Trial for Best Disease