Core Insights - Opus Genetics has initiated the first dosing in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease, marking a significant milestone for the inherited retinal disease community [1][4] - The trial aims to evaluate the safety, tolerability, and preliminary efficacy of a one-time subretinal injection of OPGx-BEST1, targeting the underlying genetic cause of Best disease [6][9] 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 [9] - The company is advancing multiple AAV-based programs, including OPGx-LCA5 and OPGx-BEST1, with a pipeline targeting various genetic mutations associated with severe retinal disorders [9] Clinical Trial Details - The OPGx-BEST1 trial is a multi-center, adaptive, open-label study designed to explore the safety and efficacy of the gene therapy in patients with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) [6] - Initial data from the trial is expected in the first quarter of 2026, with the study focusing on functional and anatomical endpoints related to visual function and retinal structure [6] Disease Background - Best disease, caused by mutations in the BEST1 gene, leads to progressive vision loss and affects an estimated 9,000 patients in the United States [2][8] - The BEST1 gene is crucial for producing bestrophin, a protein that regulates chloride ion movement in retinal cells, and its mutations can result in severe retinal conditions [7][8]
Opus Genetics Announces Dosing of First Participant in OPGx-BEST1 Phase 1/2 Gene Therapy Clinical Trial for Best Disease