Illustrative Potency Assay Examples from Approved Therapies

Sílvia Torrents; Marta Grau-Vorster; Joaquim Vives

doi:10.1007/978-3-031-30040-0_9

Illustrative Potency Assay Examples from Approved Therapies

Adv Exp Med Biol. 2023:1420:139-149. doi: 10.1007/978-3-031-30040-0_9.

Authors

Sílvia Torrents^{1

2}, Marta Grau-Vorster^{1

2}, Joaquim Vives^{3

4

5}

Affiliations

¹ Banc de Sang i Teixits, Edifici Dr. Frederic Duran i Jordà, Barcelona, Spain.
² Transfusion Medicine group, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, Barcelona, Spain.
³ Banc de Sang i Teixits, Edifici Dr. Frederic Duran i Jordà, Barcelona, Spain. jvives@bst.cat.
⁴ Musculoskeletal Tissue Engineering Group, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, Barcelona, Spain. jvives@bst.cat.
⁵ Departament de Medicina, Universitat Autònoma de Barcelona, Barcelona, Spain. jvives@bst.cat.

PMID: 37258788
DOI: 10.1007/978-3-031-30040-0_9

Abstract

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.

Keywords: Approved ATMP; CAR T-cell potency; CAR T-cell therapy; Cell therapies; Compliance; Epithelial stem cells; Gene therapies; Hematopoietic stem cells; Immunotherapy; Tissue engineering.