Opus Genetics has reported early gains in cone-mediated vision among adolescents treated with its investigational gene therapy OPGx-LCA5 for Leber congenital amaurosis type 5 (LCA5). Data from the phase 1/2 LC5A-IRD trial showed improvements in visual acuity, full-field stimulus testing, and orientation and mobility assessments at 3 months in 3 pediatric participants aged 16-17.

The pediatric group demonstrated an average visual acuity improvement of 0.3 logMAR, exceeding that of the adult cohort. One participant improved 0.7 logMAR from baseline, maintained through 3 months. Full-field testing revealed more than a one log unit improvement in cone sensitivity to both red and blue light in all treated eyes. Orientation and mobility testing also showed better object identification compared with baseline, with greater gains in the treated eye for 2 of the 3 participants.

OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. The therapy was well tolerated with no dose-limiting toxicities or serious ocular adverse events.

Durability of response has been supported by adult data, with visual acuity improvements sustained for up to 18 months. Across all 6 participants treated to date, adverse events were mild and consistent with surgical procedures. Opus plans to meet with the US Food and Drug Administration (FDA) in the fourth quarter of 2025 to discuss next steps for the program. RP