中国药物警戒 ›› 2025, Vol. 22 ›› Issue (7): 728-734.
DOI: 10.19803/j.1672-8629.20250265

• 细胞和基因治疗产品评价研究专栏 • 上一篇    下一篇

人类诱导多能干细胞质量控制及测试方法

杨怡帆1,2, 杨艳伟, 张頔1, 谢寅1, 张勇1, 陈旭林1, 高苏涛1, 耿兴超3#, 屈哲1,*   

  1. 1中国食品药品检定研究院国家药物安全评价监测中心,药品监管科学全国重点实验室,细胞及基因治疗药物质量和非临床研究与评价北京市重点实验室,北京 100176;
    2中国药科大学多靶标天然药物全国重点实验室新药筛选与药效评价中心,江苏 南京 210009;
    3中国食品药品检定研究院生物制品检定所,北京102629
  • 收稿日期:2025-04-27 出版日期:2025-07-15 发布日期:2025-07-17
  • 通讯作者: *屈哲,女,博士,研究员,药物临床前安全性评价。E-mail: quzhe@nifdc.org.cn #为共同通信作者。
  • 作者简介:杨怡帆,女,在读硕士,药物临床前安全性评价。△为并列第一作者。
  • 基金资助:
    国家重点研发计划（2024YFA1107302）

Quality Control and Test Methods of Human Induced Pluripotent Stem Cells

YANG Yifan1,2, YANG Yanwei, ZHANG Di1, XIE Yin1, ZHANG Yong1, CHEN Xulin1, GAO Sutao1, GENG Xingchao3#, QU Zhe1,*   

  1. 1National 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;
    2New Drug Screening and Pharmacodynamics Evaluation Center, State Key Laboratory of Natural Medicines, China Pharmaceutical University, Nanjing Jiangsu 210009, China;
    3Institute for Biological Products Control, National Institutes for Food and Drug Control, Beijing 102629, China
  • Received:2025-04-27 Online:2025-07-15 Published:2025-07-17

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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。

关键词: 诱导多能干细胞, 生产过程, 质量控制, 测试方法

Abstract: 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.

Key words: Induced Pluripotent Stem Cells, Production Process, Quality Control, Testing Methods

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