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

Syllabus/Topics

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 21^st 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
    

Acknowledgment
Accompanying text created by Roxy Terteryan, RKS Project Administrator, SC CTSI (atertery@usc.edu)