Usher syndrome and gene therapy for rare diseases

This episode has two parts. The first part discusses how Odylia Therapeutics is addressing rare disease, using a novel non-profit model. The second half address two rare genetic eye diseases and the approaches being designed to address them. Dr. Ashley Winslow, CEO/CSO of Odylia, describes how a non-profit is well suited to address these rare diseases, using strategies that leverage capacities in rare disease patient communities coupled to their expertise in drug discovery.