Illustrative Potency Assay Examples from Approved Therapies

Advanced therapy medicinal products (ATMP) encompass a new type of drugs resulting from the manipulation of genes, cells, and tissues to generate innovative medicinal entities with tailored pharmaceutical activity. Definition of suitable potency tests for product release are challenging in this cont...

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Bibliographic Details
Published inAdvances in experimental medicine and biology Vol. 1420; p. 139
Main Authors Torrents, Sílvia, Grau-Vorster, Marta, Vives, Joaquim
Format Journal Article
LanguageEnglish
Published United States 01.01.2023
Subjects
CAR T-cell therapy
Compliance
Tissue engineering
Immunotherapy
Cell therapies
Epithelial stem cells
Gene therapies
CAR T-cell potency
Approved ATMP
Hematopoietic stem cells
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Summary:Advanced therapy medicinal products (ATMP) encompass a new type of drugs resulting from the manipulation of genes, cells, and tissues to generate innovative medicinal entities with tailored pharmaceutical activity. Definition of suitable potency tests for product release are challenging in this context, in which the active ingredient is composed of living cells and the mechanism of action often is poorly understood. In this chapter, we present and discuss actual potency assays used for the release of representative commercial ATMP from each category of products (namely, KYMRIAH (tisagenlecleucel), Holoclar (limbal epithelial stem cells), and PROCHYMAL /RYONCIL™ (remestemcel-L)). We also examine concerns related to the biological relevance of selected potency assays and challenges ahead for harmonization and broader implementation in compliance with current quality standards and regulatory guidelines.
ISSN:0065-2598
DOI:10.1007/978-3-031-30040-0_9