OPGx-RDH12

OPGx-RDH12 is an investigational gene therapy designed to treat Leber congenital amaurosis 13 (LCA13), a severe inherited retinal dystrophy caused by mutations in the RDH12 gene. The therapy uses an adeno-associated virus (AAV) vector to deliver a functional copy of the RDH12 gene directly to retinal cells, aiming to restore protein expression and halt or slow further vision loss. Preclinical studies have focused on evaluating its ocular tolerability and potential for functional restoration in animal models, with results supporting continued development. The drug is being developed by Opus Genetics in collaboration with the University of Pennsylvania and National Resilience[1][5][6].