Gene Therapy for LCA5 Blindness Holds Gains at 24 Months

Jeff's Daily Pick. Three adults treated with Opus Genetics' OPGx-LCA5 gene therapy kept their gains in visual acuity through 24 months, the company says. Six patients in total. Phase 3 will use the same low dose, with dosing due to start in late 2026.

In the United States, Opus Genetics, a company based in Research Triangle Park, North Carolina, reported new data on October 15, 2026 from its gene therapy for Leber congenital amaurosis type 5 (LCA5), an inherited retinal disease that causes severe vision loss from childhood. Rodrigo Jorge of the Ribeirão Preto Medical School at the University of São Paulo presented the results at the Retina in Rio 2026 meeting in Brazil.

What the therapy does

Mutations in the LCA5 gene stop the retina from making lebercilin, a protein that photoreceptors need to work. OPGx-LCA5 uses an AAV8 virus to deliver a working copy of the gene to the photoreceptors, in a single injection under the retina.

The data so far

Six participants received the low dose: three adults and three adolescents. According to the company, the adults showed sustained improvements in visual acuity through 24 months. The adolescents showed restored cone mediated vision, with light sensitivity improvements reaching normal ranges through six months. Both groups had better light sensitivity than in their untreated eyes. Participants also reported everyday changes, such as recognizing family members and getting around without a cane. The press release gives no acuity scores for individual patients.

Phase 3

Based on these results, Opus moved into Phase 3 at the low dose without testing higher doses. Enrollment closed in August 2026. Patients first go through a six month run in period in which they serve as their own controls; dosing is expected in the fourth quarter of 2026, and topline six month data by the end of 2027. The primary endpoint is retinal sensitivity measured at 16 central test locations. The company says the FDA indicated that a license application could rest on the six month result, with 12 month durability data filed during review.

LCA5 disease is ultra rare, which is why the Phase 3 trial is planned for only eight participants, treated in both eyes, who serve as their own controls. For another gene therapy that splits a gene too large for one virus, see our piece on Kinea's dual AAV approach.