USH1C gene therapy

USH1C gene therapy is an investigational gene replacement treatment being developed by Odylia Therapeutics for the treatment of Usher Syndrome type 1C (USH1C). Usher Syndrome type 1C is a rare genetic disorder caused by mutations in the *USH1C* gene, which encodes the protein harmonin. This protein is critical for the structural integrity and function of the mechanosensory hair cells in the inner ear and the photoreceptor cells in the retina. The therapy aims to deliver a functional copy of the *USH1C* gene to these target tissues to restore harmonin production, thereby potentially preventing or slowing the progression of hearing loss, vestibular dysfunction, and vision loss (retinitis pigmentosa). The program is currently in the early stages of development, with specific vector details and clinical trial phases pending further disclosure.

Other names

USH1C gene replacement therapyUSH-1C gene replacement therapyUSH 1C gene replacement therapy