FDA accepts Opus Genetics' OPGx-LCA5 gene therapy into Rare Disease Evidence Principles program

Opus Genetics announced that its gene therapy candidate OPGx-LCA5 for Leber congenital amaurosis type 5 (LCA5), a rare inherited retinal disease causing early blindness, has been accepted into the FDA's Rare Disease Evidence Principles (RDEP) program. This acceptance supports early collaboration with the FDA on regulatory strategy and clinical trial design, aiming to streamline development and approval for this ultra-rare condition with no current treatments. OPGx-LCA5 is in Phase 1/2 trials showing promising vision improvements and has multiple FDA designations including RMAT. The company will work closely with the FDA to advance the therapy toward potential approval.