Core Insights - Opus Genetics has received a positive recommendation from the Independent Data Monitoring Committee (IDMC) to continue its Phase 1/2 BEST1 clinical trial (BIRD-1) for the gene therapy OPGx-BEST1 aimed at treating Best disease [1][2][3] 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 (IRDs) [7] - The company is advancing a pipeline that includes seven AAV-based programs, with OPGx-BEST1 targeting BEST1-related retinal degeneration [7] Clinical Trial Details - The BIRD-1 trial is a multi-center, adaptive, open-label, dose-exploring study evaluating the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) [5] - Treatment involves a single subretinal injection in one eye of each participant, with two dosing cohorts being explored [5] Disease Background - Best disease, or vitelliform macular dystrophy, is a rare inherited retinal condition caused by mutations in the BEST1 gene, leading to progressive vision loss and potential blindness [6] - Approximately 9,000 patients in the United States are affected by BEST1-related inherited retinal diseases [6]
Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease