Regulatory Science Symposium: “Innovations in Regenerative Medicine Products” - Session 4: Cell Therapy Manufacturing

Regulatory Science
Quality Science
Regenerative Medicine
Emerging Technologies
Cell Therapy
Good Manufacturing Practice (GMP)
Mohamed Abou-el-Enein, MD, PhD, MSPH

Associate Professor of Medicine (Clinical Scholar), Pediatrics, and Stem Cell Biology & Regenerative Medicine, Executive Director, Joint USC/CHLA Cell Therapy Program


  1. Introduction:
    1. Dr. Mohammed Abou-el-Enein
    2. Manufacturing process for the product is complicated and difficult outside of industry setting
    3. Good Manufacturing Practices (GMP) facility for Cell Therapy Products at USC
      1. Establishing GMP is an important part of translational structure of cell therapy products
      2. Research focus is Chimeric antigen receptor (CAR)-T cells
  2. Agenda
    1. Introduction to CAR-T cells
    2. Addressing key challenges in development of CAR-T cells
    3. Translational infrastructure required for cell therapy development
  3. Principles of CAR-T cells
    1. Personalized immunotherapy using patients' own T-cells genetically engineered to target tumor antigens for cancer treatment
    2. Structure
    3. CAR-T Cell Mechanism
  4. FDA-Approved CAR T Cells
  5. Value of Car T Cell Therapy
    1. Primary obstacle: High cost
  6. Roadblocks of CAR-T cell therapy (Discussion)
    1. Limited availability of long-term data – this is a factor for reimbursement bodies and health insurance companies
    2. Lack of comparison to Standard of Care
    3. Complex manufacturing of individualized therapy
  7. Long term outcomes: Meta-Analysis on Leukemia (B-ALL)
  8. CART T vs. Standard Care: Cost-Effectiveness
  9. Manufacturing Process Of CAR-T Cells
  10. Cell Therapy Manufacturing: Challenges → Response
  11. Good Manufacturing Process (cGMP)
    1. Set of standards regulated by the US Food and Drug Administration to ensure high quality, safety and efficacy of products given to patients
      1. Highly controlled and regulated process in which protocols have to be followed
    2. Example explanation of why cGMP increases cost of the whole process and how to reduce the cost by removing a part of process
  12. cGMP Costs
    1. Comparison of Berlin Facility vs US Davis Facility
    2. Cut down cost into half by optimizing manufacturing
    3. Closing the Process
    4. Progressed from Flasks & Cell Stacks to G-Rex, into Bioreactors, into now, a device called CliniMACS Prodigy
    5. Shifting from autologous to allogenic
    6. Use of non viral vectors instead of viral vectors
  13. USC/CHLA Cell Therapy Program
  14. Members in USC-CHLA CIRM Alpha Clinic
  15. Summary
  16. Questions?

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

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