Potency assays and biomarkers for cell-based advanced therapy medicinal products

Frontiers in Immunology

Immunology

Sara Nava¹  Daniela Lisini¹  Simona Frigerio¹  Chiara Pavoni²  Josée Golay²  Chiara Capelli²  Martino Introna²  Carolina Cuofano²  Andrea Biondi³  Francesco Galli⁴  Giulio Cossu⁵  Gianvito Martino⁶  Michele Quaroni⁷  Giuseppe Gaipa⁷  Valentina Colombo⁷  Patrizia Comoli⁸  Svetlana Bezukladova⁹  Paola Panina-Bordignon⁹ 

[1] Cell Therapy Production Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy;Center of Cellular Therapy “G. Lanzani”, ASST Papa Giovanni XXIII, Bergamo, Italy;Department of Pediatrics, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy;Department of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy;Division of Cell Matrix Biology & Regenerative Medicine, Faculty of Biology, Medicine and Health (FBMH), University of Manchester, Manchester, United Kingdom;Division of Cell Matrix Biology & Regenerative Medicine, Faculty of Biology, Medicine and Health (FBMH), University of Manchester, Manchester, United Kingdom;Università Vita-Salute San Raffaele, Milan, Italy;IRCCS San Raffaele Hospital, Neuroimmunology Unit, Division of Neuroscience, Milan, Italy;Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;Laboratory of Cell and Gene Therapy Stefano Verri, ASST Monza Ospedale San Gerardo, Monza, Italy;Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;Università Vita-Salute San Raffaele, Milan, Italy;IRCCS San Raffaele Hospital, Neuroimmunology Unit, Division of Neuroscience, Milan, Italy;

关键词: advanced therapy medicinal product (ATMP);    potency;    CAR (chimeric antigen receptor);    T cell therapy;    stem cell;    tissue regeneration;    biomarker;   

DOI  :  10.3389/fimmu.2023.1186224

 received in 2023-03-14, accepted in 2023-05-24,  发布年份 2023

来源: Frontiers

【 摘 要 】

Advanced Therapy Medicinal Products (ATMPs) based on somatic cells expanded in vitro, with or without genetic modification, is a rapidly growing area of drug development, even more so following the marketing approval of several such products. ATMPs are produced according to Good Manufacturing Practice (GMP) in authorized laboratories. Potency assays are a fundamental aspect of the quality control of the end cell products and ideally could become useful biomarkers of efficacy in vivo. Here we summarize the state of the art with regard to potency assays used for the assessment of the quality of the major ATMPs used clinic settings. We also review the data available on biomarkers that may substitute more complex functional potency tests and predict the efficacy in vivo of these cell-based drugs.

【 授权许可】

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Copyright © 2023 Capelli, Cuofano, Pavoni, Frigerio, Lisini, Nava, Quaroni, Colombo, Galli, Bezukladova, Panina-Bordignon, Gaipa, Comoli, Cossu, Martino, Biondi, Introna and Golay