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) co-hosted a hybrid workshop on April 9, 2026, titled “Advancing Pediatric Cell and Gene Therapy Clinical Trials.” The workshop explored 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 convened 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

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 David Wendler, MA, PhD Acting Chief, Department of Bioethics National Institutes of Health Clinical Center
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

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 

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