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The management of risk and investment in cell therapy process development: a case study for neurodegenerative disease

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journal contribution
posted on 28.06.2019, 12:54 by Sujith Sebastian, Paul C. Hourd, Amit Chandra, David Williams, Nick Medcalf
Cell-based therapies must achieve clinical efficacy and safety with reproducible and cost-effective manufacturing. This study addresses process development issues using the exemplar of a human pluripotent stem cell-based dopaminergic neuron cell therapy product. Early identification and correction of risks to product safety and the manufacturing process reduces the expensive and time-consuming bridging studies later in development. A New Product Introduction map was used to determine the developmental requirements specific to the product. Systematic Risk Analysis is exemplified here. Expected current valuebased prioritization guides decisions about the sequence of process studies and whether and if an early abandonment of further research is appropriate. The application of the three tools enabled prioritization of the development studies

Funding

This study was conducted under the UK Regenerative Medicine Platform as part of the work of the Cell Behaviour, Differentiation and Manufacturing Hub. It comprised part of the Pluripotent Stem Cell Platform (PSCP) project funded by the Medical Research Council, the Biotechnology and Biological Sciences Research Council and the Engineering and Physical Sciences Research Council (grant number MR/L012537/1). Professor Nicholas Medcalf was funded by the Engineering and Physical Sciences Research Council (grant number EP/K037099/1).

History

School

  • Mechanical, Electrical and Manufacturing Engineering

Published in

Regenerative Medicine

Volume

14

Issue

5

Pages

465–488

Citation

SEBASTIAN, S. ... et al., 2019. The management of risk and investment in cell therapy process development: a case study for neurodegenerative disease. Regenerative Medicine, 14(5), pp. 465–488.

Publisher

Future Medicine Ltd (© the Authors)

Version

VoR (Version of Record)

Publisher statement

This work is made available according to the conditions of the Creative Commons Attribution 4.0 International (CC BY 4.0) licence. Full details of this licence are available at: http://creativecommons.org/licenses/ by/4.0/

Acceptance date

08/04/2019

Publication date

2019-06-18

Copyright date

2019

Notes

This is an Open Access Article. It is published by Future Medicine under the Creative Commons Attribution 4.0 Unported Licence (CC BY). Full details of this licence are available at: http://creativecommons.org/licenses/by/4.0/

ISSN

1746-0751

eISSN

1746-076X

Language

en