Developing Gene Therapies that Work Across Mutations

One of the challenges for developing gene therapies for inherited eye diseases is that a large number of individual mutations to a gene can drive the same disease. That makes conventional gene replacement therapy a difficult approach to treat a large number of patients. Ocugen is developing gene therapies that target master regulators of genetic networks and can work across different mutations. We spoke to Arun Upadhyay, chief scientific officer and head of research and development at Ocugen, about inherited retinal diseases, the company’s platform technology that can work across different genetic mutations, and its potential applications beyond the eye. One note before we begin. Since recording this podcast, Ocugen announced that Carisma Therapeutics and Ocugen’s subsidiary OrthoCellix, entered into a definitive merger agreement to create a Nasdaq-listed, late clinical stage regenerative cell therapy company focused on orthopedic diseases. That transaction is not discussed in this interview.