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Meeting 2: Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases

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 enrollment of rare disease patients into gene therapy clinical trials in the pre-symptomatic or early symptomatic stages of their disease.  

Some rare genetic diseases can be detected in infancy or childhood, early adulthood, or later in a persons’ life, but visible decline in health and effects on quality of life may not be apparent for years or decades.  However, even among patients with the same genetic mutation, the rate of progression or severity of symptoms can vary widely and often cannot be predicted.  Caregivers for children in pre-symptomatic or early stages of a disease or adults who are in the pre-symptomatic or early stages of a disease or may be interested in participating in a gene therapy clinical trial to determine if an investigational gene therapy might prevent or meaningfully slow the progression or delay the onset of a genetic disease. 

The objective of this meeting is to understand what patients, and caregivers of patients in the pre-symptomatic or early stages of disease take into consideration when deciding whether to enroll in a gene therapy clinical trial and potentially receive an investigational gene therapy product.

Meeting Logistics and Registration

  • Date: December 4, 2024
  • Time: 11:00 a.m.– 4:30 p.m. ET
  • Location: This is a virtual public listening meeting and will be held via Zoom.
  • Registration: This meeting is free and open to the public; however, registration is required. Early registration is recommended. Please register for the event now.

Registration will close on Tuesday, December 3, at 11:59 p.m. ET. A recording and other relevant meeting materials will be posted online following the event. 

Requests to Speak or Present

During the registration process, you may indicate if you wish to speak and/or present during the public listening meeting. Speakers should indicate the session in which they would like speak or present during the meeting.  If interested in speaking about both topics, please select the session which represents your primary topic of interest during the registration process.

We will do our best to accommodate all requests. Individuals and organizations with common interests are urged to consolidate or coordinate their presentations, and request time for a joint presentation, or submit requests for designated representatives to participate. All requests to make oral presentations must have been received by November 5, 2024, at 11:59 p.m. ET. 

Based on the number of presentation requests, we will determine the amount of time allotted to each presenter and the approximate start time for each presentation. We will notify participants shortly after November 1 with additional instructions and deadlines. Please note, speakers who opt to present during this meeting can share slides, but slides are not mandatory. 

About FDA CBER

Vision: CBER uses sound science and regulatory expertise to:

  • Protect and improve public and individual health in the United States and, where feasible, globally;
  • Facilitate the development, approval of, and access to safe and effective products and promising new technologies; and
  • Strengthen CBER as a preeminent regulatory organization for biologics.

Mission: CBER’s mission is to ensure the safety, purity, potency, and effectiveness of biological products including vaccines, allergenics, blood and blood products, and cells, tissues, and gene therapies for the prevention, diagnosis, and treatment of human diseases, conditions, or injury. Through our mission, we also seek to protect the public against the threats of emerging infectious diseases and bioterrorism.

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