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

被引：10

作者：

Capelli, Chiara ^{[1
]}

Cuofano, Carolina ^{[1
]}

Pavoni, Chiara ^{[1
]}

Frigerio, Simona ^{[2
]}

Lisini, Daniela ^{[2
]}

Nava, Sara ^{[2
]}

Quaroni, Michele ^{[3
]}

Colombo, Valentina ^{[3
]}

Galli, Francesco ^{[4
]}

Bezukladova, Svetlana ^{[5
,6
]}

Panina-Bordignon, Paola ^{[5
,6
]}

Gaipa, Giuseppe ^{[3
]}

Comoli, Patrizia ^{[7
]}

Cossu, Giulio ^{[4
,5
]}

Martino, Gianvito ^{[6
,7
]}

Biondi, Andrea ^{[8
,9
]}

Introna, Martino ^{[1
]}

Golay, Josee ^{[1
]}

机构：

[1] ASST Papa Giovanni XXIII, Ctr Cellular Therapy G Lanzani, Bergamo, Italy

[2] Fdn IRCCS Ist Neurol Carlo Besta, Cell Therapy Prod Unit, Milan, Italy

[3] ASST Monza Osped San Gerardo, Lab Cell & Gene Therapy Stefano Verri, Monza, Italy

[4] Univ Manchester, Fac Biol Med & Hlth FBMH, Div Cell Matrix Biol & Regenerat Med, Manchester, England

[5] Univ Vita Salute San Raffaele, Milan, Italy

[6] IRCCS San Raffaele Hosp, Div Neurosci, Neuroimmunol Unit, Milan, Italy

[7] Fdn IRCCS Policlin San Matteo, Pediat Hematol Oncol, Pavia, Italy

[8] Fdn IRCCS San Gerardo Tintori, Dept Pediat, Monza, Italy

[9] Univ Milano Bicocca, Dept Med & Surg, Monza, Italy

来源：

FRONTIERS IN IMMUNOLOGY | 2023年 / 14卷

关键词：

advanced therapy medicinal product (ATMP); potency; CAR (chimeric antigen receptor); T cell therapy; stem cell; tissue regeneration; biomarker; EPSTEIN-BARR-VIRUS; MESENCHYMAL STROMAL CELLS; CYTOTOXIC T-LYMPHOCYTES; INDUCED KILLER-CELLS; ACUTE LYMPHOBLASTIC-LEUKEMIA; ORGAN TRANSPLANT RECIPIENTS; STEM-CELLS; DENDRITIC CELLS; PROGENITOR CELLS; ADOPTIVE IMMUNOTHERAPY;

D O I：

10.3389/fimmu.2023.1186224

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

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.

引用

页数：18

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