Core Insights - The U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Opus Genetics' investigational gene therapy OPGx-LCA5 for treating Leber Congenital Amaurosis (LCA) due to mutations in the LCA5 gene, marking a significant milestone for the company and the patient community [1][3][4] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on developing gene therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders [6] - The company's pipeline includes AAV-based investigational gene therapies targeting mutations in various genes associated with retinal diseases, including LCA and retinitis pigmentosa [6] Product Details - OPGx-LCA5 is designed to treat LCA caused by biallelic mutations in the LCA5 gene, which encodes the lebercilin protein, and is currently in a Phase 1/2 clinical trial [5][6] - The therapy utilizes an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to the outer retina, aiming to address severe vision loss in affected patients [5] Regulatory Milestones - The RMAT designation allows for expedited development and review of OPGx-LCA5, providing early interactions with the FDA and guidance on efficient development and manufacturing [4] - Opus Genetics has been invited to participate in the FDA's Initial Comprehensive Multidisciplinary RMAT Meeting and the Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) program [3]
Opus Genetics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for OPGx-LCA5 Gene Therapy Candidate