关于基因治疗药物生物分布研究检测方法的探讨

Discussions on the detection strategy and method for bio-distribution of gene therapy medicine

生物分布研究是考察药物自给药部位进入人体以后到达靶器官的情况,和伴随时间延长的代谢消除趋势,是基因治疗药物特有的实验内容。人体暴露于外源基因片段时易于发生严重危害,需要采用适宜方法考察药物基因进入体内情况以预测其潜在安全风险。当前,多个监管机构均要求在非临床阶段开展此项研究,并且在申报临床试验之前提供相关数据。本文主要概括了生物分布研究实验内容、样本种类及特点、分析方法及原理和样本检测策略,以期望指导这项研究更加合理有效地开展,和更好地服务于我国新药安全性评价领域的监管科学工作。

The aim of bio-distribution study is to investigate the performance of medicine reaching the target organs after it was administrated to the human body,as well as its metabolism and clearance process with the time extending.Bio-distribution is a particular and feature research area specific for gene therapy medicines.It could lead severe hazards when human body exposing exogenous gene segments,thus it’s necessary to adopt appropriate methods to monitor the state of gene segments of medicines inside the human body to predict potential safety risks.Currently,many regulatory authorities request to carry out bio-distribution research in preclinical study and provide relevant data and information before application in clinical trials.This article mainly generalizes the testing items,types and features of samples,analytical methods and detective strategy of bio-distribution study to provide the industry and regulatory agency with detailed and practical guidance for these kinds of researches and consequently prompt the regulation science in China in the field of preclinical evaluation and assessment.