Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center of Biotherapy, Chengdu, Sichuan 610041, China.
Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, and Collaborative Innovation Center of Biotherapy, Chengdu, Sichuan 610041, China; Key Laboratory of Drug-Targeting and Drug Delivery System of the Education Ministry, Sichuan Engineering Laboratory for Plant-Sourced Drug and Sichuan Research Center for Drug Precision Industrial Technology, West China School of Pharmacy, Sichuan University, Chengdu, Sichuan 610041, China.
Biotechnol Adv. 2020 May-Jun;40:107502. doi: 10.1016/j.biotechadv.2019.107502. Epub 2019 Dec 27.
With the improvement of gene vectors, the rise of chimeric antigen receptor T cell immunotherapy and breakthroughs in the genome editing technology, gene therapy had once again returned to the central stage of disease treatment. It had brought new choices to clinical therapy of diseases such as tumors and genetic diseases, and had changed the status quo of treatment for monogenic disorders and diffuse large B-cell lymphoma. Until August 2019, 22 gene medicines had been approved by the drug regulatory agencies from various countries, but there were few relevant reviews of combing these drugs systematically. Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed vectors. Gene therapy had gradually been accepted by the government and the public since 1980s, and have become a new and important alternative to existing treatments for human diseases in the past few years. Therefore, gene therapy drugs, with safe vectors and advanced biotechnologies, would play a greater role in the prevention and treatment of human diseases in future.
随着基因载体的改良、嵌合抗原受体 T 细胞免疫疗法的兴起和基因组编辑技术的突破，基因治疗再次回到疾病治疗的中心舞台。它为肿瘤和遗传病等疾病的临床治疗带来了新的选择，并改变了单基因疾病和弥漫性大 B 细胞淋巴瘤的治疗现状。截至 2019 年 8 月，已有 22 种基因药物获得各国药品监管机构的批准，但很少有对这些药物进行系统梳理的相关综述。因此，本综述详细总结了 1998 年至 2019 年全球批准的基因治疗药物，包括名称、适应证、批准日期、公司、载体、基因治疗药物所采用的技术和机制等。此外，根据所采用的载体对基因治疗药物进行了分类和讨论。自 20 世纪 80 年代以来，基因治疗逐渐被政府和公众所接受，近年来已成为现有人类疾病治疗方法的一种新的重要替代方法。因此，未来具有安全载体和先进生物技术的基因治疗药物将在人类疾病的预防和治疗中发挥更大的作用。
