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

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.