Advancing Pediatric Cell and Gene Therapy Clinical Trials: Scientific, Ethical, Regulatory, and Practical Considerations April 9, 2026

Workshop | Mixed

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• Date: April 9, 2026
• Time: 9:00 a.m. - 4:30 p.m. ET
• Location: FDA, White Oak Campus, The Great Room; Attend In Person or Online; Virtual: Via YouTube ; In Person: FDA White Oak Campus; 10903 New Hampshire Ave; Building 31, Room 1503; Silver Spring, MD 20993; United States

• Organized By: Center for Biologics Evaluation and Research (CBER)

The FDA’s Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products (OTP) and the Alliance for Regenerative Medicine (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 (CGTs) in pediatric populations, particularly for diseases where earlier intervention may result in greater therapeutic benefit. This includes conditions that affect both pediatric and adult populations, as well as diseases that occur primarily in children. 
FDA and ARM are convening this public workshop to discuss the following topics with relevant stakeholders:

• 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

Additional meeting materials and other updates will be posted to this webpage as they become available.

Who should attend:

This workshop is open to the public and is intended for clinicians, scientists, bioethicists, academic and industry representatives, patients, caregivers, and their advocates.

Agenda:

9:00 – 9:10 AM ET	Welcome and Introduction Vijay Kumar, MD Acting Director, Office of Therapeutic Products (OTP) Center for Biologics Evaluation and Research (CBER), FDA Mike Lehmicke, MSc Sr. Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine
9:10 – 9:35 AM ET	Ethical Considerations: Balancing Protection and Promise Tom Whitehead Co-Founder, Emily Whitehead Foundation Sharon King Chief Operating Officer, National MPS Society Moderator (invited)
9:35 – 10:40 AM ET	Panel Discussion: Regulatory and Scientific Challenges and Opportunities in Pediatric Cell and Gene Therapy (CGT) Development Lynne Yao, MD  Director, Division of Pediatric and Maternal Health Center,  Center for Drug Evaluation and Research (CDER), FDA Crystal Mackall, MD  Founding Director, Stanford Center for Cancer Cell Therapy, Stanford University Ronald J. Bartek  Co-Founder and President, Friedreich’s Ataxia Research Alliance Anne-Virginie "AV" Eggimann, MSc  Vice President/Chief Development Officer, Lilly Regenerative Medicine  Moderator: Najat Bouchkouj, MD Associate Director for Pediatrics OTP, CBER, FDA
10:40 – 11:00 AM ET	BREAK
11:00 – 1:15 PM ET	Case Studies: Real-World Examples of Pediatric CGT Development Adrenoleukodystrophy Florian Eichler, MD Director, Center for Rare Neurological Diseases, Massachusetts General Hospital Sickle Cell Disease  Lydia Pecker, MD Director of Research & Advocacy, Sickle Cell Center for Adults, Johns Hopkins University  Lupus Shaun Jackson, MD, PhD Attending Physician, Pediatric Nephrology and Rheumatology, Seattle Children’s Hospital  Mucopolysaccharidosis type I Robert Sikorski, MD, PhD Chief Medical Officer, Immusoft  Rett Syndrome Andrew Mulberg, MD Senior Vice President, Neurogene, Inc.  Inborn Errors of Immunity Fyodor Urnov, PhD Director of Therapeutic R&D, Innovative Genomics Institute Q&A Session Moderator:  Nancy Myers, JD President, Catalyst Healthcare Consulting
1:15 – 2:15 PM ET	LUNCH
2:15 – 3:15 PM ET	Panel Discussion: Prospect of Direct Benefit and Pre-Trial Data Requirements Melanie Bhatnagar, MD  Associate Director for Pediatric Education and Outreach Office of Pediatric Therapeutics (OPT), FDA Nirali Shah, MD, MHSc  Senior Investigator Pediatric Oncology Branch, National Cancer Institute (NCI) Louise R. Rodino Klapac, PhD  President, R&D and Technical Operations Sarepta Therapeutics Brett Kopelan, MA Executive Director DEBRA of America Rebecca Ahrens-Nicklas, MD, PhD  Associate Chief for Research in the Division of Human Genetics Children’s Hospital of Philadelphia Marshall Summar, MD Chief Executive Officer Uncommon Cures, LLC Moderator:  Rosa Sherafat-Kazemzadeh, MD Deputy Office Director Office of Clinical Evaluation, OTP, CBER, FDA
3:15 – 4:15 PM ET	Panel Discussion: Earliest Acceptable Disease Stage for Pediatric CGT Trial Enrollment Patroula Smpokou, MD  Director, Division of Clinical Evaluation General Medicine (DCEGM) OTP, CBER, FDA Sam Barone, MD Chief Medical Officer, Nanoscope Therapeutics Donald B. Kohn, MD  Distinguished Professor, Microbiology, Immunology and Molecular Genetics, UCLA Stem Cell Research Center Lejla Vajzovic, MD Professor of Ophthalmology, Duke University School of Medicine Kelly Brazzo  Co-Founder and CEO, CureLGMD2i Foundation Moderator: Shelby Elenburg, MD Acting Branch Chief, General Medicine Branch 1 DCEGM, OTP, CBER, FDA
4:15 – 4:30 PM ET	Closing Comments Megha Kaushal, MD, MSc Acting Deputy Director, OTP, CBER, FDA

Meeting Logistics and Registration:

This workshop 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.

To register for the workshop virtually, please use this link

To register for the workshop in-person, please use this link

• Registration for in-person attendance will remain open until 11:59 p.m. Eastern 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.
• Persons attending this conference virtually may register until the start time of the meeting.

Special Accommodations

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 OTPEvents@fda.hhs.gov 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 inquiries about in-person attendance, reach out to FDA: OTPEvents@fda.hhs.gov  
For registration and agenda inquiries, reach out to ARM: pedscgtctworkshop@catalysthcc.com 

Stay Connected

Sign up for the CBER listserv to stay up to date on all FDA CBER news and events. Find news and updates about the Alliance for Regenerative Medicine on its website: https://alliancerm.org/