The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases.

The objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes.  The meeting will be divided into two sessions.

Register for the Zoom meeting here: https://web.cvent.com/event/0b2da86e-f7d1-4288-9d11-2cb9cabe87c5/regProcessStep

Registration will close on Thursday, September 19, 2024, at 11:59 pm ET. A recording and other relevant meeting materials will be posted online following the event.