Department of Health Sciences and Technology, Samsung Advanced Institute for Health Sciences and Technology (SAIHST), Sungkyunkwan University, Seoul, Korea.
Cell and Gene Therapy Institute (CGTI), Research Institute for Future Medicine, Samsung Medical Center, Seoul, Korea.
Ann Lab Med. 2024 Jul 1;44(4):314-323. doi: 10.3343/alm.2023.0382. Epub 2024 Feb 16.
The safety and efficacy of both cell and gene therapies have been demonstrated in numerous preclinical and clinical trials. Chimeric antigen receptor T (CAR-T) cell therapy, which leverages the technologies of both cell and gene therapies, has also shown great promise for treating various cancers. Advancements in pertinent fields have also highlighted challenges faced while manufacturing cell and gene therapy products. Potential problems and obstacles must be addressed to ease the clinical translation of individual therapies. Literature reviews of representative cell-based, gene-based, and cell-based gene therapies with regard to their general manufacturing processes, the challenges faced during manufacturing, and QC specifications are limited. We review the general manufacturing processes of cell and gene therapies, including those involving mesenchymal stem cells, viral vectors, and CAR-T cells. The complexities associated with the manufacturing processes and subsequent QC/validation processes may present challenges that could impede the clinical progression of the products. This article addresses these potential challenges. Further, we discuss the use of the manufacturing model and its impact on cell and gene therapy.
细胞和基因治疗的安全性和有效性已在众多临床前和临床试验中得到证实。嵌合抗原受体 T(CAR-T)细胞疗法利用了细胞和基因治疗的技术,也为治疗各种癌症带来了巨大的希望。相关领域的进展也凸显了制造细胞和基因治疗产品所面临的挑战。为了促进个体化治疗的临床转化,必须解决潜在的问题和障碍。关于代表性的基于细胞、基于基因和基于细胞基因治疗的文献综述,涉及它们的一般制造工艺、制造过程中面临的挑战以及 QC 规范,受到了限制。我们综述了细胞和基因治疗的一般制造工艺,包括涉及间充质干细胞、病毒载体和 CAR-T 细胞的制造工艺。制造工艺和随后的 QC/验证工艺的复杂性可能带来挑战,从而阻碍产品的临床进展。本文讨论了这些潜在的挑战。此外,我们还讨论了制造模型的使用及其对细胞和基因治疗的影响。
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