Regulatory Science Symposium: “Innovations in Regenerative Medicine Products” - Session 2: Regulatory Aspects of Cell Therapy and Regenerative Medicine


  1. Agenda:
    1. Definitions
    2. FDA oversight
    3. Regenerative Medicine
    4. Expedited Development
  2. Definitions:
    1. Cell Therapy
    2. Gene Therapy
    3. Regenerative medicine
      1. Gene and cell therapies are biologics
  3. Small molecules (Drugs) vs large molecules (Biologics)
  4. Drugs
  5. Biologics
    • You test the process as much as the product.
    • High degree of variability throughout the steps.
  6. Regulations in Regenerative Medicines
  7. FDA Oversight
    1. CDER (Drugs and Therapeutic proteins)
    2. CBER (Vaccines, Blood and Biologics)
    3. Medical Devices
    4. Office of Combination Products
    5. Other Offices (Food, Radiation-Emitting Products, Animal and Veterinary, Cosmetics, Tobacco)
  8. FDA Oversight/CBER (Cellular Biology and Evolution Research)
    1. Regulates blood and blood products, vaccines tissue and tissue products, and cellular and gene therapies.
    2. Current structure: Office of Therapeutic Products (OTP) formerly the Office of Tissues and Advanced Therapies (OTAT)
  9. Guidance documents for the industry
  10. 32 guidance documents since 2007
    1. focused on therapeutic area, regulatory requirements, manufacturing, testing, and use of viral vectors, etc.
  11. Most recent guidance: “Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products” (July 2023)
  12. OTP regulated products include:
  13. Gene therapy
  14. Stem cells or stem cell-derived products
  15. Tissues
  16. Blood and Plasma Derived Products
  17. Link: Approved Cellular and Gene Therapy Products | FDA
  18. FDA's role in regulating RTMs :
  19. Responsible for lifecycle of products
    1. regulate products
    2. oversight clinical trials
    3. help in development
    4. engage stakeholders to facilitate the development
  20. Advancement in RMT
  21. Numerous investigational new drugs (INDs) for ongoing clinical studies for cell and gene therapy treatments
  22. Bespoke Gener Therapy Consortium (BGTC)
    • Partnership between NIH, FDA, and pharmaceutical companies
    • Accelerate development of gene therapies for rare diseases
  23. Requirements to qualify for RMAT
  24. RMAT established as part of 21st Century Cures Act
  25. Product has to be regenerative medicine therapy
  26. Request made concurrently with IND or as amendment to IND, but need to have clinical evidence
  27. Expedited Programs
    1. Biological License Applications (BLA) have had success
  28. FDA and Sponsor Interactions to Expedite Development
    1. Initial targeted engagement for regulatory advice on CBER products (INTERACT)
    2. CBER Advanced Technology Team (CATT)
  29. Regenerative Medicine Globally
    1. Alliance for Regenerative Medicine (2022)
    2. North America has a total of 686 institutions involved in RMT with total funding of $10.8 billion (of $12.6 billion globally)
    3. Pipeline
  30. Resources
  31. Questions

Accompanying text created by Roxy Terteryan, RKS Project Administrator, SC CTSI (


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