Cell Therapies and Tissue-Based Products: A Public Workshop on Generating Scientific Evidence to Facilitate Development

February 25, 2025

About this Course

The FDA’s Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products (OTP) hosted a virtual scientific public workshop on February 25, 2025, titled “Cell Therapies and Tissue-based Products: A Public Workshop on Generating Scientific Evidence to Facilitate Development.” The purpose of the workshop was to identify and discuss the current state of the science, development, and regulation for cellular therapies and tissue-based products.

Agenda

Welcome and Opening Remarks from FDA

• Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER), FDA
• Nicole Verdun, MD, Super Office Director, Office of Therapeutic Products (OTP), CBER, FDA

Keynote Address

• Sally Temple, PhD, Scientific Director, The Neural Stem Cell Institute

Session 1: Tissue- and Cell-Derived Therapies

• Moderator: Elizabeth Lessey-Morillon, PhD, Office of Cellular Therapy and Human Tissue CMC (OCTHT), OTP, CBER, FDA
• Development of a Biologic for Osteoarthritis
  • Katie Mowry, PhD, Vice President of Research and Development, Organogenesis
• Clinical Use of Cord Blood and Blood Cord Mesenchymal Stem Cells (MSCs) and MSC-Exosomes
  • Amanda Olson, MD, Associate Professor of Medicine, MD Anderson Cancer Center
• Neural Stem Cell Derived Biologic: Cleared IND
  • Steven Stice, PhD, Co-Founder and Chief Scientific Officer, Aruna Bio, Inc.

Session 2: Nonclinical Considerations for Cell Therapy Product Development

• Moderator: Maria Torruella Suarez, PhD, Office of Pharmacology/Toxicology, OTP, CBER, FDA
• Autologous Dopamine Neuron Replacement for Parkinson’s Disease
  • Jeanne Loring, PhD, Co-Founder, Aspen Neuroscience and Professor Emeritus, Scripps Research
• A Cell Based Therapy Trial for Huntington’s Disease
  • Leslie Thompson, PhD, Professor, University of California, Irvine
• Translating Retinal Pigment Epithelium (RPE) Biology into Disease Treatments Using Induced Pluripotent Stem (iPS) Cells
  • Kapil Bharti, PhD, Senior Investigator and Scientific Director, National Eye Institute, National Institutes of Health

Session 3: CMC Considerations for Cell Therapy Characterization

• Moderator: Thomas Finn, PhD, OCTHT, OTP, CBER, FDA
• Case Study: A Cell-based Therapy Proves Efficious, Safe, Rational, & Mechanistically-justified in an Authentitic Pre-Clinical Model of a Major Unmet Medical Need, Suggesting a Clinical Trial Should Be the Next Step
  • Evan Snyder, MD, PhD, Director of the Stem Cell Research Center and Professor, Sanford Burnham Prebys Medical Discovery Institute
• CMC Considerations for Assay Development: How Much is Enough?
  • Deborah Hursh, PhD, Principal, Hursh Cell Therapy Consulting, LLC
• CMC Considerations for Cell Therapy Characterization: The Industry Perspective
  • Jane Lebkowski, PhD, President, Regenerative Patch Technologies

Session 4: Cell Therapies for Niche Indications: Clinical Insights and Future Directions

• Moderator: Vaishali Popat, MD, Office of Clinical Evaluation, OTP, CBER, FDA
• Biologics and Other ‘Stuff’ that Gets Injected in Sports Medicine and Orthopedics
  • Blake Boggess, DO, Professor, Duke University School of Medicine; Duke Sports Medicine Team Physician
• Emerging Treatments: Cell Therapy and Synthetic Grafts for Corneal Endothelial Dysfunction
  • Ellen Koo, MD, Associate Professor of Clinical Ophthalmology, Bascom Palmer Eye Institute, University of Miami Health System
• Cell Therapy for Heart Disease
  • Eduardo Marbán, MD, PhD, Executive Director, Smidt Heart Institute, Mark S. Siegel Family Foundation Distinguished Professor

Session 5: Considerations for a Revised Risk-Based HCT/P Framework

• Moderator: Heather Lombardi, PhD, OCTHT, OTP, CBER, FDA
• Considerations for a Revised Risk-Based HCT/P Framework
  • Julie Tierney, JD, Deputy Center Director, CBER, FDA
  • Melissa A. Greenwald, MD, FAST, Chief Medical Officer, American Association of Tissue Banks

Insights and Remarks from Keynote Speaker

• Sally Temple, PhD, Scientific Director, The Neural Stem Cell Institute

visit the FDA webpage