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基于多能干细胞的细胞治疗产品的国家特定法规和国际标准化。

Country-specific regulation and international standardization of cell-based therapeutic products derived from pluripotent stem cells.

机构信息

Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Kanagawa, Japan.

Center for Regenerative Medicine, National Center for Child Health and Development, Tokyo, Japan.

出版信息

Stem Cell Reports. 2023 Aug 8;18(8):1573-1591. doi: 10.1016/j.stemcr.2023.05.003.


DOI:10.1016/j.stemcr.2023.05.003
PMID:37557074
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10444560/
Abstract

Currently, many types of cell-based therapeutic products (CTPs) derived from pluripotent stem cells (PSCs) are being developed in a lot of countries, some of which are in clinical trial stages. CTPs are classified differently in different countries and regions. The evaluation of their efficacy, safety, and quality also differs from that for conventional small-molecule drugs and biopharmaceuticals, which reflects the complex properties of living cells and unmet medical needs. Since there are no international guidelines to evaluate CTPs, including PSC-derived products, it is necessary to be aware of differences in relevant laws and regulations in different countries and regions. International consortia are organized and actively working to standardize/harmonize the evaluation methods and regulations to facilitate the development and global distribution of PSC-derived CTPs. In this paper, we outline the regulations related to PSC-derived CTPs in the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use founding regions (US, EU/UK, Japan) and introduce representative consortia working on their standardization.

摘要

目前,许多国家正在开发基于多能干细胞(PSCs)的多种细胞治疗产品(CTPs),其中一些已进入临床试验阶段。不同国家和地区对 CTPs 的分类不同,对其疗效、安全性和质量的评估也与传统的小分子药物和生物制药不同,这反映了活细胞的复杂特性和未满足的医疗需求。由于目前尚无评估 CTPs(包括 PSC 衍生产品)的国际指南,因此有必要了解不同国家和地区相关法律法规的差异。国际联盟组织起来并积极努力规范/协调评估方法和法规,以促进 PSC 衍生 CTPs 的开发和全球分布。本文概述了人用药品技术要求国际协调理事会(美国、欧盟/英国、日本)成立地区与 PSC 衍生 CTPs 相关的法规,并介绍了正在标准化方面开展工作的代表性联盟。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fba2/10444560/fd5f7a35e9c8/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fba2/10444560/fd5f7a35e9c8/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fba2/10444560/fd5f7a35e9c8/gr1.jpg

相似文献

[1]
Country-specific regulation and international standardization of cell-based therapeutic products derived from pluripotent stem cells.

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[9]
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[10]
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本文引用的文献

[1]
The consequences of recurrent genetic and epigenetic variants in human pluripotent stem cells.

Cell Stem Cell. 2022-12-1

[2]
Perspectives on the cost of goods for hPSC banks for manufacture of cell therapies.

NPJ Regen Med. 2022-9-29

[3]
Patient access to and ethical considerations of the application of the European Union hospital exemption rule for advanced therapy medicinal products.

Cytotherapy. 2022-7

[4]
Biodistribution studies for cell therapy products: Current status and issues.

Regen Ther. 2021-7-12

[5]
Non-clinical assessment of cell therapy products: the perspective from five Asian countries/regions based on regulatory guidelines and the underpinning rationales.

Cytotherapy. 2021-10

[6]
The International Stem Cell Banking Initiative (ISCBI).

Stem Cell Res. 2021-5

[7]
The Global Alliance for iPSC Therapies (GAiT).

Stem Cell Res. 2020-12

[8]
Multisite studies for validation and improvement of a highly efficient culture assay for detection of undifferentiated human pluripotent stem cells intermingled in cell therapy products.

Cytotherapy. 2021-2

[9]
Access to stem cell data and registration of pluripotent cell lines: The Human Pluripotent Stem Cell Registry (hPSCreg).

Stem Cell Res. 2020-6-27

[10]
A Manually Curated Database on Clinical Studies Involving Cell Products Derived from Human Pluripotent Stem Cells.

Stem Cell Reports. 2020-8-11

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