基因疗法的临床开发：头三十年及未来发展

Lapteva Larissa, Purohit-Sheth Tejashri, Serabian Mercedes, Puri Raj K

Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.

Division of Cellular and Gene Therapies, Center for Biologics Evaluation and Research, Office of Tissues and Advanced Therapies, U.S. Food and Drug Administration, Silver Spring, MD, USA.

Mol Ther Methods Clin Dev. 2020 Oct 10;19:387-397. doi: 10.1016/j.omtm.2020.10.004. eCollection 2020 Dec 11.

In the past three decades the field of gene therapy has made remarkable progress, surging from mere laboratory experiments to Food and Drug Administration (FDA)-approved products that bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. Herein, we review the evolution of the gene therapy clinical research landscape and describe the gene therapy product development programs evaluated by the FDA in Investigational New Drug applications received in 1988-2019. We also discuss the clinical development programs of the first six oncolytic and gene therapy products approved in the United States.

在过去三十年中，基因治疗领域取得了显著进展，从单纯的实验室实验迅速发展到获得美国食品药品监督管理局（FDA）批准的产品，这些产品为那些之前对其严重病症没有治疗选择的患者带来了疾病负担的显著减轻。在此，我们回顾基因治疗临床研究领域的发展历程，并描述FDA在1988 - 2019年收到的研究性新药申请中评估的基因治疗产品开发项目。我们还讨论了美国批准的首批六种溶瘤和基因治疗产品的临床开发项目。