The biopharmaceutical company, Ocugen Inc., has announced that the FDA has accepted their Investigational New Drug (IND) application to start a human clinical trial with OCU400. This is a modifier gene therapy for retinitis pigmentosa (RP) caused by mutations in NR2E3 and Rhodopsin. The therapy will target nuclear hormone receptors (NHRs). NHRs control many functions in the retina so they can potentially address many different gene mutations, which means they can be a therapy for different types of retinal diseases. Most gene therapies only target one individual gene mutation at a time. The Ocugen therapy, if it works, could treat many different types of gene mutations. The clinical trial will start in early 2022 and will be enrolling patients with mutations in NR2E3 and RHO genes.
What this means for Usher syndrome: Vision loss in Usher syndrome is caused by retinitis pigmentosa (RP), thus OCU400 is a potential treatment that could help restore visual function in Usher patients. Clinical trials in humans will be starting in 2022, and if found effective could be closer to becoming an available treatment.