Overview of the international regulatory policies for stem cell therapy products and its implications for China's regulatory system development

doi:10.3877/cma.j.issn.2095-1221.2025.02.008

Abstract

Abstract:

Stem cell therapy， as representatives of new quality productive forces in the field of biomedical， have high development potential and broad application prospects.In view of the particularity and complexity of stem cell therapy， governments and regulatory authorities of various countries have strengthened their supervision over the field of stem cell therapy， striving to promote the healthy development of the field while ensuring patient safety.In this context， it is theoretical and practical significant to study and discuss the domestic and international regulatory policies and technical specifications for stem cell therapy.This article comprehensively reviews the current regulatory policies in major countries and regions worldwide， systematically analyze relevant guidelines， discusses the challenges faced in stem cell therapy regulation，and proposes recommendations for research on regulatory system.This paper aims to provide valuable references for the safe， effective and compliant application of stem cell therapy.

Key words: Stem cell therapy, Regulatory, Technical guidelines

Ying Huang, Tiantian Hou, Ziyi Jin, Xingchao Geng. Overview of the international regulatory policies for stem cell therapy products and its implications for China's regulatory system development[J]. Chinese Journal of Cell and Stem Cell(Electronic Edition), 2025, 15(02): 120-126.

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URL: https://zhxbygxbzz.cma-cmc.com.cn/EN/10.3877/cma.j.issn.2095-1221.2025.02.008

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References 33

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名称	发布时间
Human Gene Therapy Products Incorporating Human Genome Editing； Guidance for Industry	2024 年1 月
包含人类基因组编辑的基因治疗产品；行业指南
Potency Assurance for Cellular and Gene Therapy Products； Draft Guidance for Industry	2023 年12 月
细胞和基因治疗产品的效价保证；行业指南草案
Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products； Draft Guidance for Industry	2023 年7 月
细胞和基因治疗产品的生产变更和可比性研究；行业指南草案
Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial； Guidance for Industry	2022 年11 月
在早期临床试验中研究细胞或基因治疗产品的多个版本
Chemistry，Manufacturing， and Control（ CMC） Information for Human Gene Therapy Investigational New Drug Applications（ INDs）；Guidance for Industry	2020 年1 月
基因治疗新药研究申请（ IND）的化学、生产和控制（ CMC）信息；行业指南
Long Term Follow-up After Administration of Human Gene Therapy Products； Guidance for Industry	2020 年1 月
基因治疗产品用药后的长期随访；行业指南
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions； Guidance for Industry	2019 年2 月
针对严重疾病的再生医学疗法的快速审评计划；行业指南
Regulatory Considerations for Human Cells，Tissues，and Cellular and Tissue-Based Products：Minimal Manipulation and Homologous Use；Guidance for Industry and Food and Drug Administration Staff Updated	2017 年12 月
人体细胞、组织以及细胞组织产品的监管考虑因素：最小化操作和同源使用；更新的行业和FDA 员工指南
Deviation Reporting for Human Cells，Tissues，and Cellular and Tissue-Based Products Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR Part 1271；Guidance for Industry	2017 年9 月
仅根据《公共卫生服务法》第 361 节和《美国联邦法规汇编》第 21 篇第 1271 部分进行监管的人体细胞、组织以及基于细胞和组织的产品的偏差报告；行业指南
Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products；Guidance for Industry	2015 年6 月
细胞和基因治疗产品早期临床试验设计注意事项；行业指南
Guidance for Industry：BLA for Minimally Manipulated，Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System	2014 年3 月
行业指南：用于影响造血系统疾病患者造血和免疫重建的微操作、非亲缘异体胎盘/ 非脐带血的BLA
IND Applications for Minimally Manipulated， Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System - Guidance for Industry and FDA Staff	2014 年3 月
针对患有影响造血系统疾病的患者进行造血和免疫重建的微操作、非亲缘异体胎盘/ 非脐带血 IND 申请-- 行业和 FDA 员工指南
Guidance for Industry： Preclinical Assessment of Investigational Cellular and Gene Therapy Products	2013 年11 月
行业指南：细胞和基因治疗研究产品的临床前评估
Guidance for Industry： Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage	2011 年11 月
行业指南：用于修复或替换膝关节软骨的产品 IDE 和 IND 的准备工作
Guidance for Industry：Potency Tests for Cellular and Gene Therapy Products	2011 年1 月
行业指南：细胞和基因治疗产品的效力测试
Guidance for Industry： Cellular Therapy for Cardiac Disease	2010 年1 月
行业指南：细胞疗法治疗心脏病
Guidance for Industry： Considerations for Allogeneic Pancreatic Islet Cell Products	2009 年9 月
行业指南：异体胰岛细胞产品的注意事项
Guidance for FDA Reviewers and Sponsors：Content and Review of Chemistry，Manufacturing， and Control（ CMC）Information for Human Somatic Cell Therapy Investigational New Drug Applications（ INDs）	2008 年4 月
FDA 评审员和申办者指南：人体细胞疗法新药研究申请（ IND）的化学、生产和控制（ CMC）信息的内容和审查
Eligibility Determination for Donors of Human Cells，Tissues，and Cellular and Tissue-Based Products；Guidance for Industry	2007 年8 月
人体细胞、组织以及细胞和组织产品捐赠者的资格确定；行业指南
Guidance for Industry：Guidance for Human Somatic Cell Therapy and Gene Therapy	1998 年3 月
行业指南：人类体细胞疗法和基因疗法指南

名称	发布时间
Human Gene Therapy Products Incorporating Human Genome Editing； Guidance for Industry	2024 年1 月
包含人类基因组编辑的基因治疗产品；行业指南
Potency Assurance for Cellular and Gene Therapy Products； Draft Guidance for Industry	2023 年12 月
细胞和基因治疗产品的效价保证；行业指南草案
Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products； Draft Guidance for Industry	2023 年7 月
细胞和基因治疗产品的生产变更和可比性研究；行业指南草案
Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial； Guidance for Industry	2022 年11 月
在早期临床试验中研究细胞或基因治疗产品的多个版本
Chemistry，Manufacturing， and Control（ CMC） Information for Human Gene Therapy Investigational New Drug Applications（ INDs）；Guidance for Industry	2020 年1 月
基因治疗新药研究申请（ IND）的化学、生产和控制（ CMC）信息；行业指南
Long Term Follow-up After Administration of Human Gene Therapy Products； Guidance for Industry	2020 年1 月
基因治疗产品用药后的长期随访；行业指南
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions； Guidance for Industry	2019 年2 月
针对严重疾病的再生医学疗法的快速审评计划；行业指南
Regulatory Considerations for Human Cells，Tissues，and Cellular and Tissue-Based Products：Minimal Manipulation and Homologous Use；Guidance for Industry and Food and Drug Administration Staff Updated	2017 年12 月
人体细胞、组织以及细胞组织产品的监管考虑因素：最小化操作和同源使用；更新的行业和FDA 员工指南
Deviation Reporting for Human Cells，Tissues，and Cellular and Tissue-Based Products Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR Part 1271；Guidance for Industry	2017 年9 月
仅根据《公共卫生服务法》第 361 节和《美国联邦法规汇编》第 21 篇第 1271 部分进行监管的人体细胞、组织以及基于细胞和组织的产品的偏差报告；行业指南
Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products；Guidance for Industry	2015 年6 月
细胞和基因治疗产品早期临床试验设计注意事项；行业指南
Guidance for Industry：BLA for Minimally Manipulated，Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System	2014 年3 月
行业指南：用于影响造血系统疾病患者造血和免疫重建的微操作、非亲缘异体胎盘/ 非脐带血的BLA
IND Applications for Minimally Manipulated， Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System - Guidance for Industry and FDA Staff	2014 年3 月
针对患有影响造血系统疾病的患者进行造血和免疫重建的微操作、非亲缘异体胎盘/ 非脐带血 IND 申请-- 行业和 FDA 员工指南
Guidance for Industry： Preclinical Assessment of Investigational Cellular and Gene Therapy Products	2013 年11 月
行业指南：细胞和基因治疗研究产品的临床前评估
Guidance for Industry： Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage	2011 年11 月
行业指南：用于修复或替换膝关节软骨的产品 IDE 和 IND 的准备工作
Guidance for Industry：Potency Tests for Cellular and Gene Therapy Products	2011 年1 月
行业指南：细胞和基因治疗产品的效力测试
Guidance for Industry： Cellular Therapy for Cardiac Disease	2010 年1 月
行业指南：细胞疗法治疗心脏病
Guidance for Industry： Considerations for Allogeneic Pancreatic Islet Cell Products	2009 年9 月
行业指南：异体胰岛细胞产品的注意事项
Guidance for FDA Reviewers and Sponsors：Content and Review of Chemistry，Manufacturing， and Control（ CMC）Information for Human Somatic Cell Therapy Investigational New Drug Applications（ INDs）	2008 年4 月
FDA 评审员和申办者指南：人体细胞疗法新药研究申请（ IND）的化学、生产和控制（ CMC）信息的内容和审查
Eligibility Determination for Donors of Human Cells，Tissues，and Cellular and Tissue-Based Products；Guidance for Industry	2007 年8 月
人体细胞、组织以及细胞和组织产品捐赠者的资格确定；行业指南
Guidance for Industry：Guidance for Human Somatic Cell Therapy and Gene Therapy	1998 年3 月
行业指南：人类体细胞疗法和基因疗法指南

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