Therapeutic Programs
We are early in our journey but we are making effective strides for rare disease and vision loss. Newly launched this year, our Usher Syndrome gene therapy for patients with vision loss caused by USH1C mutations is the newest program to join the Odylia portfolio. Staying true to our mission and core values, the USH1C program was developed in partnership with a community of experts and two different patient groups focused on developing treatments for Usher Syndrome. USH1C joins the Odylia portfolio alongside the RPGRIP1 program, a gene therapy to treat patients with vision loss caused by mutations in RPGRIP1. PTC Therapeutics licensed the RPGRIP1 gene therapy program in June of 2019.
Our Partners
