The first session of the CMT Research Foundation’s Gene Therapies Demystified series gave attendees the building blocks: what gene therapy is, why CMT is a compelling target, and what the science suggests about its potential. The second session moved from the foundational to the practical, tackling clinical trial design.
The final installment, Policy, Access, and the Road Ahead, examines the federal landscape surrounding gene therapy development. As regulatory agencies, Congress, and the administration navigate a period of significant change, the decisions being made right now will have lasting consequences for patients with CMT and rare disease communities broadly.
John Feore, Director of Health Policy & Advocacy for the Institute for Gene Therapies, will take stock of where things stand at the FDA, HHS, and on Capitol Hill, mapping the policy developments that are opening new doors for innovation and those that threaten to close them. It will also explore a side of drug development that rarely gets public attention: how organizations like IGT work alongside patient advocacy groups to improve access, shape policy, and ensure that the patient voice is part of the conversation from the beginning.
For patients, caregivers, and anyone invested in the future of gene therapy for CMT, this session offers an essential look at the forces beyond the lab that determine whether a promising treatment ever reaches the people who need it.
The third installment of Gene Therapies Demystified takes place on July 16 at 7pm Eastern.
