The Do’s and Don’ts of Managing a Pivotal Gene Therapy Trial
Summary: Gene therapies have been exploding in popularity, with big and small players lining up scores of new programs looking to create entirely new standard of care models for a wide variety of diseases. But what does it take to de-risk a pivotal, registration-worthy gene therapy program? What are the pitfalls to avoid and the opportunities in creating more efficient designs that are more likely to get a nod from regulators?
Deborah Wild, Chief of Staff and VP of Corporate Development at Catalent; Kathy Reape, Chief Medical Officer at Spark Therapeutics; Bobby Gaspar, President of Research and Chief Scientific Officer at Orchard Therapeutics; Michael Binks, Vice President of Clinical Research at Pfizer; Wing Yen Wong, Vice President of Clinical Science at BioMarin; and Endpoints News editor John Carroll present strategies to bring a gene therapy to market.