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

VernacularTitle:腺相关病毒载体基因治疗药物生物分析技术及药代/药效动力学研究进展
Author: Jianwei REN ¹ ; Xin ZHENG ; Xiaohong HAN
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1. 中国医学科学院北京协和医院临床药理研究中心疑难重症及罕见病全国重点实验室国家药监局药物临床研究与评价重点实验室创新药物临床PK/PD北京市重点实验室,北京 100730
Keywords: adeno-associated virus; bioassays; gene therapy; biodistribution; shedding
From: JOURNAL OF RARE DISEASES 2024;3(3):350-357
CountryChina
Language:Chinese
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.