Aug 03, 2026 —
Opus Genetics announced that the last patient has been enrolled in its registrational Phase 3 clinical trial evaluating OPGx-LCA5, an investigational AAV8 gene therapy for LCA5-associated inherited retinal disease.
Completion of enrollment marks a key development milestone for the program, which was accepted into the U.S. Food and Drug Administration’s Rare Disease Evidence Principles, or RDEP, program in May 2026. The Phase 3 study was designed in collaboration with the FDA to support a potential Biologics License Application, or BLA.
OPGx-LCA5 is designed to treat a form of Leber congenital amaurosis, or LCA, caused by biallelic mutations in the LCA5 gene. LCA5 encodes lebercilin, a protein important for retinal function. LCA5-associated inherited retinal disease is an early-onset, severe inherited retinal dystrophy that can lead to profound vision loss.
The therapy uses an adeno-associated virus 8, or AAV8, vector to deliver a functional LCA5 gene to the outer retina through a one-time subretinal administration. Opus Genetics is developing the therapy as a potential first approved treatment for patients with LCA5-associated inherited retinal disease.
The registrational Phase 3 trial evaluates the safety and efficacy of OPGx-LCA5 in patients with genetically confirmed disease. Consistent with the company’s RDEP discussions with the FDA, the study includes evidence-generation approaches suited to an ultra-rare disease, including a six-month run-in period in which patients serve as their own control.
Participants are currently completing the run-in period. Opus Genetics expects to initiate dosing in the fourth quarter of 2026 and report topline six-month efficacy data by the end of 2027.
The company said it may be able to submit a BLA based on six-month efficacy data, with 12-month durability data provided during FDA review. This regulatory strategy reflects the program’s alignment with the FDA through the RDEP framework.
OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy, or RMAT, designations from the FDA. Opus Genetics also continues to expect that the therapy may qualify for a Rare Pediatric Disease Priority Review Voucher if approved.
The completion of enrollment advances OPGx-LCA5 toward a critical dosing and data-readout period. If successful, the program could represent an important step for AAV-based retinal gene therapy in ultra-rare inherited retinal diseases with no approved treatment options.
