Eisenman Daniel, Swindle Scott
Biosafety Services, Advarra, Inc., Columbia, Maryland, USA.
Appl Biosaf. 2022 Sep 1;27(3):191-197. doi: 10.1089/apb.2022.0020. Epub 2022 Sep 14.
The US regulatory environment is evolving to accommodate a boom in gene therapy research. The 2019 version of the National Institutes of Health (NIH) Guidelines on Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines) lacks an appendix providing specific guidance for Institutional Biosafety Committee (IBC) review of clinical trials.
As the field matures, the burden of Federal oversight for clinical trials of investigational products containing recombinant or synthetic nucleic acid molecules is shifting toward the Food and Drug Administration (FDA). This report summarizes recent FDA guidance documents on shedding and considerations for environmental impact assessments highlighting key points pertinent to IBC review.
This report helps biosafety professionals understand the evolving regulatory framework for gene therapy products. Knowledge of the guidance documents discussed in this report will assist biosafety professionals in addressing issues pertaining to shedding and environmental impact during IBC review of clinical trials.
美国监管环境正在不断演变，以适应基因治疗研究的蓬勃发展。美国国立卫生研究院（NIH）2019年版的《涉及重组或合成核酸分子的研究指南》（NIH指南）缺少一个附录，该附录可为机构生物安全委员会（IBC）对临床试验的审查提供具体指导。
随着该领域的成熟，联邦政府对含有重组或合成核酸分子的研究性产品临床试验的监督负担正转向食品药品监督管理局（FDA）。本报告总结了FDA最近关于脱落以及环境影响评估考量的指导文件，突出了与IBC审查相关的要点。
本报告有助于生物安全专业人员理解基因治疗产品不断演变的监管框架。了解本报告中讨论的指导文件将有助于生物安全专业人员在IBC对临床试验的审查过程中解决与脱落和环境影响相关的问题。
