Practice and Reflection on Non-clinical Safety Evaluation of AAV drugs

¹ Department of Toxicology, Zhengzhou University School of Public Health, Zhengzhou, Henan, 450001, China
² Beijing Zhaoyan New Drug Research Center Co., Ltd, Beijing, 100176, China

^a huangxinyu@gs.zzu.edu.cn
^b wgl18503849031@163.com
^c wangning@joinn-lab.com
^d zhangqiao@ zzu.edu.cn
^* Corresponding author: zhangsucai@joinn-lab.com

Abstract

Due to the rapid development of biomedical technology, new high-tech therapeutic methods have also developed rapidly, among which gene therapy products as a representative of therapeutic drugs are booming and constantly iterated. In the process of drug development, non-clinical safety evaluation is a crucial part, which can provide preliminary exploration for subsequent clinical trials. In the past few years, there has been a significant rise in the FDA's approval of novel gene therapy medications. However, it is important to address certain safety concerns that have emerged during non-clinical trials. This paper mainly discusses the considerations of non-clinical safety evaluation of gene therapy drugs with AAV as an example and the concerns of AAV drugs in non-clinical evaluation.