Opus Genetics Doses First Pediatric Patient in OPGx-LCA5 Gene Therapy Trial

Opus Genetics has reached a key milestone in its Phase 1/2 clinical trial evaluating OPGx-LCA5, its investigational gene therapy for Leber congenital amaurosis (LCA). The company announced that the first pediatric patient has been dosed, marking a significant step toward treating this inherited retinal disease at an earlier stage. Initial data from the pediatric cohort is expected by Q3 2025.

Early clinical results from the first three adult patients have demonstrated meaningful visual improvement, starting as early as one month post-treatment, with durability confirmed at 12 months. Opus plans to present these findings at a major medical conference in Q2 2025. Additionally, a Type D meeting with the FDA is scheduled for March to discuss Phase 3 trial design and potential registrational endpoints.

CEO George Magrath emphasized the importance of early intervention for pediatric patients, highlighting the therapy’s potential to preserve or restore vision before disease progression. The ongoing trial evaluates OPGx-LCA5’s safety and efficacy through various vision function tests, including Multi-Luminance Orientation and Mobility Test (MLoMT), Full-Field Stimulus Testing (FST), and microperimetry.

Opus Genetics, in collaboration with the University of Pennsylvania, continues to work closely with the medical and patient communities to advance this groundbreaking gene therapy, offering hope for individuals affected by LCA.