Opus Genetics, Inc. announced in a press release that the last patient has been enrolled in its registrational phase 3 clinical trial evaluating OPGx-LCA5, the company’s investigational gene therapy for LCA5-associated inherited retinal disease.

Completion of enrollment follows the program’s acceptance into the US Food and Drug Administration (FDA) Rare Disease Evidence Principles (RDEP) program in May 2026. The phase 3 study was designed in collaboration with the FDA to support a potential biologics license application (BLA) for what could become the first approved therapy for patients living with LCA5-associated inherited retinal disease, the company said in the press release. 

The  phase 3 trial is evaluating the safety and efficacy of a one-time subretinal administration of OPGx-LCA5 in patients with genetically confirmed LCA5-associated inherited retinal disease. Consistent with Opus Genetics' RDEP discussions with the FDA, the company said the study incorporates evidence-generation approaches appropriate for an ultra-rare disease, including a 6-month run-in period in which patients serve as their own control. Participants are currently completing the run-in period, and the company said it expects to initiate dosing of OPGx-LCA5 in the fourth quarter of 2026, with topline data expected by the end of 2027.

OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy designations from the FDA and has been accepted into the FDA’s RDEP program.

The company said it continues to expect that OPGx-LCA5 may qualify for a Rare Pediatric Disease Priority Review Voucher upon approval. RP