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]

Core Viewpoint - Opus Genetics, Inc. is advancing its gene therapy programs for inherited retinal diseases (IRDs) and will present findings at several scientific conferences in May 2025 [1] Presentation Details - The company will present at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting on May 13, 2025, focusing on MERTK gene therapy in RCS rats [2] - At the American Ophthalmological Society (AOS) Annual Meeting from May 15-17, 2025, the company will discuss endpoints for the first interventional gene therapy clinical study in BEST Disease [3] - An oral presentation on utilizing VR guided testing for visual function assessment is scheduled for May 29, 2025 [7] Research Highlights - Retinitis pigmentosa (RP) affects 1 in 3,000 to 1 in 7,000 people, with 3% of cases linked to mutations in the MERTK gene [5] - OPGx-MERTK, an investigational gene therapy, showed effective preservation of photoreceptors and retinal function in a rat model after a single bilateral subretinal injection [5] - OPGx-BEST1 is being developed for BEST1-related inherited retinal diseases, with preclinical studies showing restoration of the RPE-photoreceptor interface in treated canines [6] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on gene therapies for IRDs and other ophthalmic disorders [8] - The pipeline includes AAV-based investigational gene therapies targeting mutations in LCA, BEST1, and retinitis pigmentosa [8] - The company plans to initiate a Phase 1/2 trial of OPGx-BEST1 in 2025, aiming for preliminary data by Q1 2026 [6]