Workshop: Advancing Pediatric Cell and Gene Therapy Clinical Trials

Scientific, Ethical, Regulatory, and Practical Considerations

About the Event

The FDA’s Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products (OTP) and ARM are co-hosting a hybrid workshop on April 9, 2026, titled “Advancing Pediatric Cell and Gene Therapy Clinical Trials.” The workshop will explore the issues surrounding clinical trials for cell and gene therapies in pediatric populations, particularly for diseases where earlier intervention may result in greater therapeutic benefit. Discussion topics include:

• Challenges and potential solutions in pediatric clinical trials for CGT products

• Data and information needed to enroll pediatric patients in CGT clinical trials, including interpretations of the prospect of direct benefit

• Appropriate timing to enroll pediatric patients in CGT trials

• Considerations for enrollment of children at presymptomatic and early disease stages

April 9, 2026

FDA White Oak Campus | 10903 New Hampshire Ave, Building 31, Silver Spring, MD 20903

This is a hybrid workshop

In-person and virtual attendance options are available. Register below.

Click here to view the agenda & speaker bios

Who Should Attend

Logistics

This workshop is open to the public and is intended for clinicians, scientists, bioethicists, academic and industry representatives, patients, caregivers, and their advocates. It will be held in-person on the FDA White Oak Campus and in a virtual format via YouTube. Registration is required for virtual and in-person attendance. Upon registration, registrants will receive a confirmation email with additional information.

Registration

In-person registration is based on space availability, with priority given to early registrants. Please be prepared to provide complete contact information for each attendee, including name, email address, and affiliation when registering. Registration is required for both in-person and virtual attendance. In-person and virtual registrants will receive an email confirmation after they register with additional workshop details.

How to Register

Registration for in-person attendance will remain open until 11:59 p.m. Eastern Standard Time on Monday, April 6th, 2026. Early registration is recommended because seating is limited; therefore, FDA may limit the number of participants from each organization. If time and space permit, onsite registration on the day of the public meeting (April 9, 2026) will be provided beginning at 9:00 a.m. We will post a notice on the meeting webpage if registration for in-person attendance closes before the day of the public meeting. Campus visitor information will be sent to in-person attendees prior to the workshop.

Click here to register for the workshop in-person

Virtual registration will be available until the start time of the meeting.

Click here to register for the workshop virtually

Special Accomodations

The FDA will make every effort to accommodate persons with physical disabilities or special needs at public meetings. If you require accommodations due to a disability, email [email protected] at least 7 days before the workshop. The FDA White Oak campus and FDA shuttle are generally accessible to people with physical disabilities.

Visiting the FDA Campus

Entrance for meeting attendees (non-FDA employees) is through Building 1. All attendees (FDA and non-FDA) meeting in-person will be required to pass through routine security screening. Laptops, tablets, cell phones, keys, car fobs, coins and other metal items must be removed from pockets and put in a bin or tray for X-ray screening.

Visitors to White Oak campus can use public transportation (Metro train and bus, commuter trains and buses) or taxi and ridesharing services. Limited visitor parking is available. Please allow at least 30 minutes to get from the visitor's parking lots to Building 1 and extra time to accommodate the security check-in. After passing through security screening, attendees will be directed to the workshop room in Building 31.

For additional parking and security information, please refer to Public Meeting Information, Getting to the FDA, and Visitor Parking and Campus Map.

Contact

For questions regarding visiting the FDA campus, please email: [email protected]

For registration and other questions, please email ARM: [email protected]