基因修饰细胞治疗产品的生物分析策略与方法学介绍

摘要/Abstract

摘要： 基因修饰细胞治疗产品是指经基因修饰、并按照药品监管相关法规进行研发和注册申报的活细胞产品，主要包括基因修饰的免疫细胞、干细胞以及其他类型细胞的治疗产品。此类治疗产品既有细胞产品自身特点，也有基因治疗产品的潜在风险，生物分析策略要考虑这两方面的特点。恰当设计并执行临床生物分析的检测方法，开展充分的生物分析研究将有助于明确基因修饰细胞治疗产品的有效性与安全性、并最终确立所开发产品的获益风险比，其重要性是不言而喻的。本文从药代（细胞）动力学、药效学与免疫原性的研究上综述了细胞治疗产品的生物分析的策略和实操。

关键词: font-size:medium, ">生物分析；细胞治疗；药代（细胞）动力学；免疫原性

Abstract: Genetically modified cell therapy products refer to living cell products that have been genetically-modified and are developed for marketing in accordance with relevant regulations. They mainly include genetically modified immune cells， stem cells， and other cell-derived products. While genetically modified cell therapy products have characteristics of the cell product， they carry potential risks of gene therapy because of their gene modification nature. Bioanalytical strategies should take these elements into consideration. This requires properly designed and implemented bioanalytical methods for clinical studies. Adequate bioanalysis is therefore critical to collect data and information for the evaluation of efficacy， safety， and finally the benefit-risk ratio of the gene-modified cell products， and to provide evidence to support clinical trial design and risk control strategies. This review summarizes bioanalytical strategies and practices of cell therapy products from pharmacokinetic (cellular kinetics)， pharmacodynamic， and immunogenicity evaluation perspectives.

Key words: font-size:medium, ">Bioanalysis； Cell therapy； Pharmacokinetics (Cellular kinetics)； Immunogenicity

中图分类号:

张贺峰, 梁宇, 刘颖, 邹灵龙, . 基因修饰细胞治疗产品的生物分析策略与方法学介绍[J]. 中国药物评价, 2023, 40(5): 369-376.

ZHANG He-feng, LIANG Yu, LIU Ying, ZOU Ling-long, . Introduction of Bioanalytical Strategies and Methodologies for Genetically Modified Cell Therapy Products[J]. CHINESE JOURNAL OF DRUG EVALUATION, 2023, 40(5): 369-376.

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