The 4th Annual Gene Therapy for Rare Disorders Europe is dedicated to overcoming manufacturing, clinical, and commercialization challenges drug developers face when delivering gene therapies to market.
Considerations for Selection of NovelEndpoints and Appropriate Controls
• Discussing some of the creative manners gene therapy developers have used to optimise clinical trial design
• Collecting the patient and caregiver perspective as a valuable source of information
• Understanding an individual’s trajectory to inform treatment expectations
Samantha Parker, Senior Vice President & Chief Patient Access Officer, Lysogene