Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells.
ATMP
Cell-based therapy
biodistribution
blood outgrowth endothelial cells
gene-based therapy
human Alu sequences
method validation
preclinical and non-clinical development
qPCR
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
11 Sep 2020
11 Sep 2020
Historique:
received:
13
03
2020
accepted:
27
05
2020
entrez:
9
7
2020
pubmed:
9
7
2020
medline:
9
7
2020
Statut:
epublish
Résumé
The investigation of the biodistribution profile of a cell-based medicinal product is a pivotal prerequisite to allow a factual benefit-risk assessment within the non-clinical to clinical translation in product development. Here, a qPCR-based method to determine the amount of human DNA in mouse DNA was validated according to the guidelines of the European Medicines Agency and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. Furthermore, a preclinical worst-case scenario study was performed in which this method was applied to investigate the biodistribution of 2 × 10
Identifiants
pubmed: 32637449
doi: 10.1016/j.omtm.2020.05.029
pii: S2329-0501(20)30116-9
pmc: PMC7327859
doi:
Types de publication
Journal Article
Langues
eng
Pagination
176-188Informations de copyright
© 2020 The Authors.
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