Department of Clinical Neuroscience and Wellcome-MRC Cambridge Stem Institute, John van Geest Centre for Brain Repair, Forvie Site, Robinson Way, Cambridge CB2 0QQ, UK.
ElevateBio, Cambridge, MA 02142, USA.
Stem Cell Reports. 2023 Aug 8;18(8):1599-1609. doi: 10.1016/j.stemcr.2022.11.019. Epub 2022 Dec 22.
Developing cellular therapies is not straightforward. This Perspective summarizes the experience of a group of academic stem cell investigators working in different clinical areas and aims to share insight into what we wished we knew before starting. These include (1) choosing the stem cell line and assessing the genome of both the starting and final product, (2) familiarity with GMP manufacturing, reagent validation, and supply chain management, (3) product delivery issues and the additional regulatory challenges, (4) the relationship between clinical trial design and preclinical studies, and (5) the market approval requirements, pathways, and partnerships needed.
开发细胞疗法并非易事。本观点总结了一组在不同临床领域工作的学术干细胞研究人员的经验,旨在分享我们在开始之前希望了解的见解。这些包括 (1) 选择干细胞系并评估起始和最终产品的基因组,(2) 熟悉 GMP 制造、试剂验证和供应链管理,(3) 产品输送问题和额外的监管挑战,(4) 临床试验设计与临床前研究之间的关系,以及 (5) 市场批准要求、途径和所需的合作伙伴关系。
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