Opus Genetics announces one-year results on OPGx-LCA5

Opus Genetics (IRD) announced one-year results from adult patients treated in the ongoing Phase 1/2 Study of its lead gene therapy candidate OPGx-LCA5. These results were presented yesterday at the 2025 annual meeting of the Association for Research in Vision and Ophthalmology, ARVO, taking place May 4 – 7, 2025 in Salt Lake City, Utah. The presentation, entitled “Recovery of Cone-Mediated Vision in a Severe Ciliopathy after Gene Augmentation: One Year Results of a Phase I/II Trial for LCA5-LCA,” was delivered by Dr. Tomas Aleman of the Scheie Eye Institute, University of Pennsylvania. Highlights of 12-month Phase 1/2 Study Results: The goal of this clinical trial is to evaluate the safety and preliminary efficacy results of subretinal gene therapy with OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. The results presented comprised three adult patients, all of whom received subretinal injections in a single eye of up to 300 microl of low dose OPGx-LCA5. Each patient had severe disease at baseline, with limited but detectable photoreceptors and disease that had progressed to the central retina. A further two adolescent patients have now also been treated with promising preliminary data that were not included in this presentation.