Opus Genetics has announced preliminary one-month data from the first pediatric patient enrolled in its phase 1/2 clinical trial of OPGx-LCA5, an investigational gene therapy for inherited retinal disease caused by mutations in the LCA5 gene. The 16-year-old patient, who received a single subretinal injection in February, showed a meaningful improvement in visual function and no treatment-related adverse events, according to the company.

This update follows earlier positive results from adult patients in the same trial, where improvements persisted for at least one year. A second pediatric patient has been dosed, and full enrollment of the 3-patient pediatric cohort is expected by the second quarter of 2025. Initial data from all 3 are anticipated in the third quarter of the year.

Opus reports that it also held a Type D meeting with the US Food and Drug Administration to discuss a registrational trial that could begin in 2026. The proposed design would include as few as 19 patients and use a virtual reality–based functional vision test as the primary endpoint.

OPGx-LCA5 is delivered using an AAV8 vector and aims to restore the function of the LCA5 gene, which encodes the lebercilin protein. There are currently no approved therapies for this form of Leber congenital amaurosis. RP