Core Insights - Opus Genetics has initiated dosing of the first pediatric patient in its Phase 1/2 trial for OPGx-LCA5, a gene therapy for Leber congenital amaurosis (LCA), with initial data expected by Q3 2025 [1] - The trial has shown early clinical proof of concept, with significant visual improvement observed in adult patients as early as one month post-treatment, and new 12-month data will be presented in Q2 2025 [2][3] - An FDA meeting is scheduled for March 2025 to discuss the design and endpoints for a pivotal Phase 3 trial for OPGx-LCA5 [1][3] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on developing gene therapies for inherited retinal diseases and other ophthalmic disorders [7][8] - The company’s pipeline includes investigational gene therapies targeting various genetic mutations associated with retinal diseases, with OPGx-LCA5 being the most advanced program [8] Clinical Trial Details - The Phase 1/2 trial is designed to evaluate the safety and preliminary efficacy of OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene [4] - Efficacy endpoints include functional vision measurements using Multi-Luminance orientation and Mobility Test (MLoMT), Full-Field Stimulus Testing (FST), and microperimetry [4] Future Developments - The company plans to share new 12-month data from adult patients treated with OPGx-LCA5 at a major medical conference in Q2 2025 [2][5] - The ongoing collaboration with the University of Pennsylvania is crucial for the advancement of the OPGx-LCA5 program [3]
Opus Genetics Announces Updates on OPGx-LCA5 Clinical Program