摘要
目的探讨人类诱导多能干细胞(Human Induced Pluripotent Stem Cells,hiPSC)疗法临床转化面临的挑战及其质量控制要求,为细胞治疗产品安全生产提供参考。方法通过文献调研,综述hiPSC疗法研发现状及生产流程各阶段的技术挑战,参考最新法规指南概述其质量控制要求。结果hiPSC展现出广阔的应用前景,但尚无基于hiPSC的疗法获批上市,主要障碍在于其复杂生产流程各环节的技术挑战。质量控制与产品表征是产品研发的核心要素,虽无统一标准,但对hiPSC关键质量属性已形成普遍共识。结论明确并实施基于共识的质量控制要求对于克服hiPSC疗法转化瓶颈至关重要,深入理解生产流程各环节的质量控制点,将有助于推动hiPSC衍生细胞治疗产品的安全性和有效性评估,加速其临床转化和监管审批。
Objective To explore the challenges to clinical translation of human induced pluripotent stem cell(hiPSC)therapies and define the quality control requirements in order to provide references to regulation of related products.Methods Based on literature review,the current R&D of hiPSC therapies was outlined and technical hurdles in each stage of production were identified.Quality control standards were summarized out of recent guidelines.Results While hiPSC-based therapies promised wide applications,no clinical-grade product had been approved for marketing.The major obstacles arose from technical challenges encountered in the complicated process of production.Quality control and product characterization were critical to the development and commercialization of these products.Despite the lack of a uniform standard,there was consensus regarding critical quality attributes of hiPSC-derived cell therapies.Conclusion A uniform quality control standard is essential to overcome translational barriers.Keen insights into each element of quality control during the process of production will facilitate the assessment of safety/efficacy of hiPSC-derived products,thus accelerating clinical translation and regulatory approval.
作者
杨怡帆
杨艳伟
张頔
谢寅
张勇
陈旭林
高苏涛
耿兴超
屈哲
YANG Yifan;YANG Yanwei;ZHANG Di;XIE Yin;ZHANG Yong;CHEN Xulin;GAO Sutao;GENG Xingchao;QU Zhe(National Center for Safety Evaluation of Drugs,National Institutes for Food and Drug Control,State Key Laboratory of Drug Regulatory Science,Beijing Key Laboratory of Quality Control and Non-clinical Research and Evaluation for Cellular and Gene Therapy Medicinal Products,Beijing 100176,China;New Drug Screening and Pharmacodynamics Evaluation Center,State Key Laboratory of Natural Medicines,China Pharmaceutical University,Nanjing Jiangsu 210009,China;Institute for Biological Products Control,National Institutes for Food and Drug Control,Beijing 102629,China)
出处
《中国药物警戒》
2025年第7期728-734,共7页
Chinese Journal of Pharmacovigilance
基金
国家重点研发计划(2024YFA1107302)。
关键词
诱导多能干细胞
生产过程
质量控制
测试方法
Induced Pluripotent Stem Cells
Production Process
Quality Control
Testing Methods
