Gene Therapy: The Long Horizon and Broad Challenges

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? What are the different ways that value manifests for patients, payers and society? This panel will feature in-depth interviews of global leaders and advocates in the rare disease space.