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Regulatory Considerations in Trial Design for Cell and Gene Therapies
Session Chair(s)
Kanwaljit Singh, MD, MBA, MPH
Executive Director, International Neonatal Consortium
Critical Path Institute, United States
Cell and gene therapies have enormous potential to address unmet medical need. The application of validated clinical trials design tools along with biomarkers to assess safety and endpoints can help address regulatory uncertainty.
Learning Objective : Discuss regulatory considerations for cell and gene therapies including the use of data modeling in clinical trial design and the application of biomarkers both for safety assessment and measures of outcome; Discuss how to plan for meeting regulatory requirements and be able to incorporate these plans into your company’s drug development plans.
Speaker(s)
Regulatory Considerations for Development of Pediatric Cell and Gene Therapies: FDA Update
Najat Bouchkouj, MD
FDA, United States
Associate Director for Pediatrics (Acting), Office of Clinical Evaluation
Ethical, Clinical, and Timing Considerations for Administering Cell and Gene Therapies in Neonates and Early Childhood
Paulla Dennis
Fortrea, United States
Senior Director, Rare Diseases, Advanced Therapies, and Pediatrics
Considerations Related to Safety and Efficacy Biomarkers in C> (Path from Bench-to-Bedside for a Cell and Gene Therapy Drug Product)
Stephanie Cherqui, PhD
University of California San Diego, United States
Director, UC San Diego Gene Therapy Initiative
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