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Lessons learnt, and still to learn, in first in human stem cell trials.

Accepted version
Peer-reviewed

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Type

Article

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Authors

Barker, Roger A 
Carpenter, Melissa 
Jamieson, Catriona HM 
Murry, Charles E 
Pellegrini, Graziella 

Abstract

Developing cellular therapies is not straightforward. This Perspective summarizes the experience of a group of academic stem cell investigators working in different clinical areas and aims to share insight into what we wished we knew before starting. These include (1) choosing the stem cell line and assessing the genome of both the starting and final product, (2) familiarity with GMP manufacturing, reagent validation, and supply chain management, (3) product delivery issues and the additional regulatory challenges, (4) the relationship between clinical trial design and preclinical studies, and (5) the market approval requirements, pathways, and partnerships needed.

Description

Keywords

stem cell therapy, stem cells, translation, Humans, Stem Cells, Cell- and Tissue-Based Therapy, Cell Line

Journal Title

Stem Cell Reports

Conference Name

Journal ISSN

2213-6711
2213-6711

Volume Title

Publisher

Elsevier BV
Sponsorship
Wellcome Trust (203151/A/16/Z)
Wellcome Trust (203151/Z/16/Z)
National Institute for Health and Care Research (IS-BRC-1215-20014)
Medical Research Council (MC_PC_17230)