美国的监管环境正在不断演变，以适应即将到来的基因治疗研究热潮。
The United States' Regulatory Environment Is Evolving to Accommodate a Coming Boom in Gene Therapy Research.
作者信息
Eisenman Daniel
机构信息
Advarra, Research Triangle Park, NC, USA.
出版信息
Appl Biosaf. 2019 Sep 1;24(3):147-152. doi: 10.1177/1535676019854866.
INTRODUCTION
A dramatic increase in the number of clinical trials involving gene-modified cell therapy and gene therapy is taking place. The field is on the verge of a boom, and the regulatory environment is evolving to accommodate the growth.
DISCUSSION
This commentary summarizes the current state of the field, including an overview of the growth. The United States (US) regulatory structure for gene therapy will be summarized, and the evolution of the oversight structure will be explained.
CONCLUSION
The gene therapy field has recently produced its first FDA-approved therapeutics and has a pipeline of other investigational products in the final stages of clinical trials before they can be evaluated by the FDA as safe and effective therapeutics. As research continues to evolve, so must the oversight structure. Biosafety professionals and IBCs have always played key roles in contributing to the safe, evidence-based advancement of gene therapy research. With the recent regulatory changes and current surge in gene therapy research, the importance of those roles has increased dramatically.
引言
涉及基因修饰细胞疗法和基因疗法的临床试验数量正在急剧增加。该领域正处于蓬勃发展的边缘，监管环境也在不断演变以适应其增长。
讨论
本评论总结了该领域的当前状况，包括增长概况。将概述美国基因疗法的监管结构，并解释监管结构的演变。
结论
基因疗法领域最近产生了首个获得美国食品药品监督管理局（FDA）批准的疗法，并且还有一系列其他研究产品正处于临床试验的最后阶段，之后才能由FDA评估其作为安全有效的疗法。随着研究不断发展，监管结构也必须如此。生物安全专业人员和机构生物安全委员会（IBC）在推动基因疗法研究安全、基于证据的进展方面一直发挥着关键作用。随着近期的监管变化以及当前基因疗法研究的激增，这些作用的重要性已大幅提高。
Zotero 插件