Science-based assessment of source materials for cell-based medicines: report of a stakeholders workshop

Author:

Stacey Glyn1,Andrews Peter2,Asante Curtis3,Barbaric Ivana2,Barry Jaqueline4,Bisset Louise5,Braybrook Julian6,Buckle Robin7,Chandra Amit8,Coffey Peter910,Crouch Sharon11,Driver Philip12,Evans Amanda13,Gardner John14,Ginty Patrick15,Goldring Christopher16,Hay David C17,Healy Lyn18,Hows Anna19,Hutchinson Claire16,Jesson Helen20,Kalber Tammy21,Kimber Sue22,Leathers Roland23,Moyle Sarah24,Murray Trish25,Neale Michael10,Pan David7,Park B Kevin16,Rebolledo Raul Elgueta26,Rees Ian5,Rivolta Marcelo N2,Ritchie Allan26,Roos Eric J27,Saeb-Parsy Kourosh28,Schröder Bernd29,Sebastian Sujith30,Thomas Angela31,Thomas Robert J20,Turner Marc32,Vallier Ludovic33,Vitillo Loriana10,Webster Andrew34,Williams David20

Affiliation:

1. International Stem Cell Banking Initiative, 2 High Street, Barley, Hertfordshire, SG8 8HZ, UK

2. Department of Biomedical Sciences, Centre for Stem Cell Biology, University of Sheffield, Sheffield, South Yourkshire, S10 2TN, UK

3. Centre for Stem Cells & Regenerative Medicine, King's College London, Guy's Campus, London, SE1 9RT, UK

4. Cell & Gene Therapy Catapult, London, SE1 9RT, UK

5. Medicines & Healthcare Products Regulatory Agency, London, E14 4PU, UK

6. LGC Ltd, National Measurement Laboratory, Teddington, TW11 0LY, UK

7. Medical Research Council, London, WC2B 4AN, UK

8. Yposkesi, 91100 Corbiel-Essonnes, France

9. University of California, Neuroscience Research Institue, Santa Barbara, CA, 93106, USA

10. Institute of Opthalmology, University College London, London, WC1E 6BT, UK

11. Financial & Business Services, University of Nottingham, Nottingham, NG7 2RD, UK

12. Royal Society of Chemistry, London, W1J 0BA, UK

13. University of Cambridge/NHS Blood & Transplant, Long Road, CB2 0PT, Cambridge, UK

14. School of Social Sciences, Monash University, Victoria, Australia.

15. Regulatory Affairs, Cell and Gene Therapy Catapult, London, SE1 9RT, UK

16. Department of Molecular and Clinical Pharmacology, University of Liverpool, Liverpool, L69 3GE, UK

17. University of Edinburgh, MRC Centre for Regenerative Medicine, Edinburgh, EH16 4UU, UK

18. Francis Crick Institute, London, NW1 1AT, UK

19. Miltenyi Biotec, Bisley, GU24 9DR, UK

20. Centre for Biological Engineering, Loughborough University, Loughborough, LE11 3TU, UK

21. Centre for Advanced Biomedical Imaging, University College London, WC1E 6BT, UK

22. Division of Cell Matrix Biology and Regenerative Medicine; Director EPSRC/MRC in Regenerative Medicine, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester, M13 9PT, UK

23. Thermo Fisher Scientific, CH-4123 Allschwil, CH, USA

24. Clinical Biomanufacturing Facility, Oxford University, Oxford, OX3 7JT, UK

25. Department of Physiology, University of Liverpool, Liverpool, L69 3GE, UK

26. Allan Ritchie Medical Device Consulting, Harrogate, HG1 1BX, UK

27. Thermofisher, Boston, MA, USA

28. Department of Surgery, University fo Cambridge, Cambridge Biomedical Campus, Cambridge, CB2 0QQ, UK

29. Miltenyi Biotec GmbH, 51429 Bergisch Gladbach, Nordrhein-Westfalen, Germany

30. School of Bioscience, University of East Anglia, Norwich Research Park, Norwich, NR4 7TJ, UK

31. College of Medicine and Veterinary Medicine, University of Edinburgh, Edinburgh, EH16 4TJ, UK

32. Scottish National Blood Transfusion Service, Edinburgh, EH14 4BE, UK

33. Wellcome–Medical Research Council Cambridge Stem Cell Institute and Department of Surgery, University of Cambridge, Cambridge UK

34. SATSU, Department of Sociology, University of York, York, YO10 5DD, UK

Abstract

Human pluripotent stem cells (hPSCs) have the potential to transform medicine. However, hurdles remain to ensure safety for such cellular products. Science-based understanding of the requirements for source materials is required as are appropriate materials. Leaders in hPSC biology, clinical translation, biomanufacturing and regulatory issues were brought together to define requirements for source materials for the production of hPSC-derived therapies and to identify other key issues for the safety of cell therapy products. While the focus of this meeting was on hPSC-derived cell therapies, many of the issues are generic to all cell-based medicines. The intent of this report is to summarize the key issues discussed and record the consensus reached on each of these by the expert delegates.

Publisher

Future Medicine Ltd

Subject

Embryology,Biomedical Engineering

Reference25 articles.

1. UK Regenerative Medicine Platform (UK RMP). www.ukrmp.org.uk/

2. Quantification of biological variation in blood-based therapy - a summary of a meta-analysis to inform manufacturing in the clinic

3. Comparability: manufacturing, characterization and controls, report of a UK Regenerative Medicine Platform Pluripotent Stem Cell Platform Workshop, Trinity Hall, Cambridge, 14–15 September 2015

4. Roslin. http://roslincells.com/latest-news/2013/9/11/cell-therapy-catapult-roslin-cells-to-create-clinical-grade.html

5. National Institute for Biological Standards and Control. www.nibsc.org/science_and_research/advanced_therapies/uk_stem_cell_bank/cell_lines/approved_by_the_bank.aspx

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