Patient enrollment complete in phase 3 trial of OPGx-LCA5 for LCA5-associated inherited retinal disease

Opus Genetics Inc. announced in a recent press release that the last patient has been enrolled in its phase 3 registrational, or pivotal, trial to evaluate OPGx-LCA5, a gene therapy that is under investigation for treating Leber congenital amaurosis type 5 (LCA5)-associated inherited retinal disease.

This trial is evaluating the safety and efficacy of OPGx-LCA5 administered as a one-time subretinal treatment of patients with an LCA5-associated inherited retinal disease that was confirmed genetically. OPGx-LCA5 uses an adeno-associated virus 8 vector to precisely deliver a functional LCA5 gene to the outer retina. Dosing is expected to begin in the fourth quarter of 2026, and the topline data are expected to be announced by the end of 2027, according to the press release.

The company explained that completion of patient enrollment represents a significant milestone in the development of OPGx-LCA5 and follows the program’s acceptance into the US 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 for what could become the first approved therapy for patients with LCA5-associated inherited retinal disease.

George Magrath, MD, Chief Executive Officer of Opus Genetics, explained, “With enrollment completed, we remain on track to initiate dosing in the fourth quarter of 2026, report topline data by the end of 2027, and continue advancing OPGx-LCA5 to bring the first potential treatment to patients with an LCA5-associated inherited retinal disease.”

In addition to the acceptance into the RDEP program, OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy designations from the FDA. The company continues to expect that OPGx-LCA5 may qualify for a Rare Pediatric Disease Priority Review Voucher upon approval, representing a potentially significant strategic asset.

Phase 1/2 clinical trial of OPGx-LCA5

In September 2025, the company announced that the treatment yielded positive 3-month data in the pediatric cohort of the ongoing OPGx-LCA5-1001 trial evaluating OPGx-LCA5.

Three patients age 16 to 17 years were treated, all of whom had severe baseline visual impairment. The patients received one subretinal injection of OPGx-LCA5. The results from all patients showed improvements in the visual acuity, full-field stimulus testing; and multi-luminance orientation and mobility test.One patient underwent microperimetryand showed early signs of improved fixation stability.
The company noted that the pediatric results provide evidence that OPGx-LCA5 can potentially restore cone-mediated vision in teenagers with profound vision loss. “These outcomes, alongside observed durable improvements observed in adults out to 18 months, give us confidence in the potential for OPGx-LCA5 to deliver meaningful and lasting benefit to patients,” Magrath stated.

Ramiro Maldonado, MD, a retina specialist commented on this investigative treatment. “This is a real milestone for LCA5-associated retinal disease, an ultra-rare condition where patients rarely even get the chance to join a trial. The encouraging signals from the phase 1/2 data make this phase 3 completion especially exciting. Great to see Opus Genetics investing in such a rare gene, and I’m looking forward to the results.He is Associate Professor of Ophthalmology, Director of both the Retina Machine Learning Laboratory and the IRD Fellowship, Duke Center for Ophthalmic Genetics, Duke Pediatric Retina and Duke Adult Medical Retina, Duke University, Durham, NC.