Gene therapy: The long horizon and broad challenges

4:30 PM - 5:30 PM, Tuesday, June 9, 2020 ・ Room 8
What if patients were not patients anymore? For companies developing gene therapies for rare diseases, the goal is to develop therapies that cure the disease with a single dose. If they succeed, they'll all but eliminate the need for lifelong intervention and cause a fundamental shift in the role of advocacy groups and other hallmarks of rare disease communities. So, how does that work in practice? What challenges and opportunities do such companies face? What does “value” mean, and what are the different ways that value manifests for patients, payers and society? This panel will dive into those issues with in-depth interviews with global leaders and advocates in the rare disease space: Mark Rothera, President and CEO of Orchard Therapeutics, Jill Quigley, J.D., Chief Operating Officer of Passage Bio, and Maria Kefalas, Founder of The Calliope Joy Foundation, a nonprofit organization committed to supporting families and championing research in the area of leukodystrophy.
CEO and Founder
Endpoints News
Chief Operating Officer
Passage Bio
President and CEO
Orchard Therapeutics