Advances in Bioanalytical Techniques and Pharmacokinetics/Pharmacodynamics Research of Adeno-Associated Viral Vector Gene Therapy Drugs

Abstract

Recently, adeno-associated virus(AAV)vectors have been a hotspot in gene therapy and have shown great potential for rare diseases therapy, due to their low pathogenicity, mild immunogenicity and high tissue specificity. The safety and efficacy of AAV vectors, which are essentially infectious viruses, require new bioanalytical techniques to assess. At present, the relevant industry guidelines have specified the needs for the assessment of biodistribution, shedding, and immunogenicity, but the requirements related to bioassay are limited. In this paper, we reviewed the bioanalytical techniques and pharmacokinetics/pharmacodynamics(PK/PD)research of AAV vectors and their challenges from four aspects: biodistribution, shedding, immunogenicity and PK/PD research, with the hope of providing some reference for the clinical development of AAV vectors gene therapy products.

Keywords

• adeno-associated virus
• bioassays
• gene therapy
• biodistribution
• shedding