October 01, 2025

Gene Therapies for Inherited Retinal Diseases

Genetic testing has become the standard of care for monogenetic IRD, and retina specialists have had to make continual efforts to stay up to date in what is arguably the most rapidly evolving field of ophthalmology, the field of retinal genetics and gene therapy. This brief article will share updates from a selection of trials highlighting the following IRDs: retinitis pigmentosa (RP), focusing on augmentation gene therapy for RPGR-associated X-linked RP (XLRP) and a gene-agnostic optogenetics approach for RP, Stargardt disease, Leber congenital amaurosis (LCA), and X-linked retinoschisis (XLRS).

Retinal Physician