May 05, 2026 —
Opus Genetics announced that its investigational LCA5 gene therapy program, OPGx-LCA5, has been accepted into the U.S. FDA’s Rare Disease Evidence Principles (RDEP) program, supporting the company’s regulatory strategy for a potential pivotal Phase 3 pathway. OPGx-LCA5 is being developed for Leber congenital amaurosis type 5 (LCA5), an ultra-rare inherited retinal disease caused by mutations in the LCA5 gene that can lead to early-onset, progressive vision loss and severe visual impairment or blindness in childhood.
The FDA’s RDEP program is designed to support development of therapies for ultra-rare genetic diseases, particularly where patient populations are too small for traditional large clinical trials. Through the program, the FDA will work closely with Opus Genetics on clinical trial design, evidence generation, and strategies to demonstrate clinical benefit in a small patient population. The program may also support the use of a single adequate and well-controlled study with confirmatory evidence as part of the substantial evidence package.
OPGx-LCA5 uses an AAV8 vector to deliver a functional copy of the LCA5 gene to the outer retina. The therapy is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania. Reported data from pediatric participants showed large gains in cone-mediated vision, while adult participants demonstrated durable improvements in cone sensitivity and visual function out to 18 months. The therapy has remained well tolerated, with no ocular serious adverse events or dose-limiting toxicities reported. In addition to RDEP acceptance, OPGx-LCA5 has received FDA Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy designations.
