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.

中文翻译：

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制造细胞和基因疗法：临床转化的挑战。


细胞和基因疗法的安全性和有效性已在大量临床前和临床试验中得到证明。嵌合抗原受体 T (CAR-T) 细胞疗法利用细胞和基因疗法技术，也显示出治疗各种癌症的巨大前景。相关领域的进步也凸显了制造细胞和基因治疗产品时面临的挑战。必须解决潜在的问题和障碍，以简化个体疗法的临床转化。对代表性的基于细胞、基于基因和基于细胞的基因疗法的文献综述关于其一般制造工艺、制造过程中面临的挑战以及质量控制规范是有限的。我们回顾了细胞和基因疗法的一般制造流程，包括涉及间充质干细胞、病毒载体和 CAR-T 细胞的流程。与制造过程和随后的质量控制/验证过程相关的复杂性可能会带来阻碍产品临床进展的挑战。本文解决了这些潜在的挑战。此外，我们讨论了制造模型的使用及其对细胞和基因治疗的影响。