Illustrative Potency Assay Examples from Approved Therapies

Overview

Journal Adv Exp Med Biol

Specialties Biology
General Medicine

Date 2023 May 31

PMID 37258788

Authors

Silvia Torrents

Marta Grau-Vorster

Joaquim Vives

Affiliations

Soon will be listed here.

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.

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