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临床试验中的多能干细胞衍生疗法:2025年更新

Pluripotent stem-cell-derived therapies in clinical trial: A 2025 update.

作者信息

Kirkeby Agnete, Main Heather, Carpenter Melissa

机构信息

Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW) and Department of Neuroscience, Faculty of Health and Medical Sciences, University of Copenhagen, 2200 Copenhagen, Denmark; Department of Experimental Medical Sciences, Wallenberg Center for Molecular Medicine (WCMM) and Lund Stem Cell Center, Lund University, 221 84 Lund, Sweden.

HOYA Consulting (ReGenMed Solutions), Stockholm, Sweden.

出版信息

Cell Stem Cell. 2025 Jan 2;32(1):10-37. doi: 10.1016/j.stem.2024.12.005.

Abstract

Since the first derivation of human pluripotent stem cells (hPSCs) 27 years ago, technologies to control their differentiation and manufacturing have advanced immensely, enabling increasing numbers of clinical trials with hPSC-derived products. Here, we revew the landscape of interventional hPSC trials worldwide, highlighting available data on clinical safety and efficacy. As of December 2024, we identify 116 clinical trials with regulatory approval, testing 83 hPSC products. The majority of trials are targeting eye, central nervous system, and cancer. To date, more than 1,200 patients have been dosed with hPSC products, accumulating to >10 clinically administered cells, so far showing no generalizable safety concerns.

摘要

自27年前首次获得人类多能干细胞(hPSCs)以来,控制其分化和制造的技术有了巨大进步,使得越来越多使用hPSC衍生产品的临床试验得以开展。在此,我们综述了全球范围内干预性hPSC试验的情况,重点介绍了有关临床安全性和有效性的现有数据。截至2024年12月,我们确定了116项获得监管批准的临床试验,涉及83种hPSC产品。大多数试验针对眼睛、中枢神经系统和癌症。迄今为止,已有超过1200名患者接受了hPSC产品给药,累计临床给药细胞数超过10个,目前尚未发现普遍的安全问题。

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