Lessons from the Oregon Trail: Pioneering Gene Therapy in the Real World
Monday, June 03, 2024
Breakout Session
Cell and Gene Therapy and Genome Editing
31AB
Gene therapies hold the potential to transform – and even cure – severe genetic diseases and offer new hope for patients and families. Yet moving these promising results from the confines of a clinical trial setting into the “real world” is not for the faint of heart. Delivering one-time therapies – as opposed to chronic, lifelong treatments – requires a different approach from manufacturers, CDMOs, health systems, payers, and patients, and overcoming these challenges is essential for the field to deliver on its long-awaited promise to patients.
Hear firsthand from pioneers who've played pivotal roles in delivering the first gene therapies to patients in a commercial setting. The discussion will also include patient perspective on the gene therapy treatment journey.
Moderator
Speakers
Medical Director, Blood and Marrow Transplant Program
Children's Hospital of Philadelphia