translating cell therapies: academic versus industry model

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  1. 1. Translating cell therapies: Academic versus Industry model Alexey Bersenev, MD, PhD Advanced Cell Therapy Laboratory, Yale-New Haven Hospital, Yale University The BioProcessing Summit, Boston, Aug. 4, 2015
  2. 2. Academia vs. industry clinical trials global trends Key differences between academic and industry models Gaps and solutions Outline of the talk:
  3. 3. Global trends: sponsorship of clinical trials
  4. 4. Global trends: Total number of MSC trials
  5. 5. Global trends: Total number of trials, involved adipose-derived cells
  6. 6. Is industry in favor of allo- model? NO! Total number of registered clinical trials
  7. 7. Measuring output: published results of clinical studies in regenerative medicine 2014 sample (n=116)
  8. 8. focus on first-in-human, pilot studies early stages of development delivery: cell therapy as procedure favored model auto? favored medical innovation regulatory path diseases with low prevalence low scale no attention to pharmacoeconomics, relies on efficacy boost focus on commercialization and technology transfer delivery: cell therapy as mass- produced drug favored model allo? all phases of trials under FDA diseases with high prevalence large scale pharmacoeconomics will determine product development Academic versus industry cell models key differences: academic industry
  9. 9. Medical Research Personalized Medicine First-In-Human Serum Cell Culture Patient File/ Binder Scale Out Closed System Cost of Goods Automation Large Scale Bioprocess Batch/ Lot Unit Operations Academia vs. Industry cultural/ language difference: Academia Industry
  10. 10. developer FDA local IRB FACT AABB Traditional academic and industrial pathways CAP HCT/P biologic/ drug 361 351 No GMP cGMP need for cell culture/ gene modifications
  11. 11. Booming of stem cell and maturity of cell-based technologies on research side ready for translation! Clinical resources in big academic hospitals pioneering physician are eager to be the first Technological tools for clinical cell processing are available Pharma and Biotech are reluctant to enter the field on early stages The need for academic GMP cell therapy facilities:
  12. 12. Academic GMP cell therapy facilities: Trend: increase number of academic facilities AABM Association of Academic Biologics Manufacturers ~ 45 registered, mostly in US AGORA: ATMP GMP Open Access Research Alliance - 8 centers listed EATRIS: European Infrastructure for Translational Medicine ATMP Platform 14 GMP facilities in EU Estimated total # of facilities in US 50-100 and EU few hundreds
  13. 13. Usually not a subject of FDA inspection not always follow strict GMP standards Scale and capacity: much smaller than industry Acting as a hospital core facility must to accommodate all types cell/gene therapy trials Usually under-funded data not publishable, so no return Academic GMP facilities key differences:
  14. 14. Talking of scale: Academia Industry
  15. 15. Centralized vs. decentralized cell manufacturing facilities cGMPcGMP decentralized centralized collection/ processing OR/ APH closed system/ automation OR/ APH release delivery collection delivery processing release
  16. 16. Licensing > Tech Transfer Contract manufacturing Validation studies Academic GMP Industry interactions:
  17. 17. Key to success in tech transfer: 1. Academics must understand needs of industry for successful commercialization and regulatory compliance 2. Industry to leverage the knowledge of academics
  18. 18. Academic GMP - more gaps: Lack of QC, QA and Regulatory Research-like mindset Training Funding: The cost of GMP compliance is underestimated in academia
  19. 19. Advice for academics: Invest time in developing cell therapy programs in academia training, GMP facilities, QA/ regulatory affairs teams Start pre-clinical design and Phase 1 with pharmacoeconomics analysis think of cost- effectiviness, COGS, BLA, reimbursement Utilize (1) translational centers to help and (2) industry consultants Get involved in industry collaborations and tech transfers huge experience!
  20. 20. Advice for industry: It will be slow > be patient Educate academics for your own benefit Invest early > product/ process development. Phase 2 could be too late Utilize academics more for validation studies


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