Adeno-associated virus (AAV)-based gene therapies (GT) have shown promise for the treatment of many diseases, including rare diseases with unmet medical needs. In...

2023年2月27日 · A rapidly maturing therapeutic platform — adeno-associated virus (AAV) gene therapy — holds great promise for many rare diseases. AAV gene therapies use a modified version of a virus called adeno-associated virus to deliver a working copy of a defective gene into the cells relevant for a given disease.

2024年12月1日 · Three major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: 1) engineering vectors to increase transgene expression; 2) aligning interests of the health system with costs and challenges for the pharmaceutical industry; and 3) refining patient eligibility criteria and endpoint defin...

In this review, we summarize PK studies for the AAV-based GT products approved so far by U.S. Food and Drug Administration and European Medicines Agency. We then comparatively review the analytical methods employed to evaluate AAV vectors’ biodistribution and shedding of approved GT products.

2024年1月12日 · AAV vectors emerge as promising tools for HCC therapy due to their non-immunogenic nature, efficient cell entry, and prolonged gene expression. While AAV-mediated GT demonstrates promise across diverse diseases, the current absence of ongoing clinical trials targeting HCC underscores untapped potential in this context.

2022年9月8日 · A better understanding of the immunogenic properties of, and the immune pathways activated by, an AAV GT may help to optimize the immunosuppressive treatment during clinical development.

2024年4月29日 · 基于腺相关病毒（Adeno-associated virus, AAV）的基因治疗（Gene therapy, GT）产品能够有效地递送目的基因并替代丢失或缺陷的基因，为患者的治疗带来希望。然而，目前对于临床前和临床试验中分析方法的研究有限，这可能阻碍基因治疗产品开发的成功。在开 …

2023年12月20日 · Although mild in many patients, hepatotoxicity may affect efficacy of gene therapy (GT), lead to acute liver injury and death [2,3,4, 5•, 6•]. This has resulted in termination of some clinical trials of rAAV vector-based GT [6•].

2018年10月7日 · Herein, we discuss considerations in the design of a comprehensive preclinical safety program for AAV-based GT prior to administration in humans. Gene therapy (GT) is a therapeutic intervention to genetically alter or modify living cells using either viral or nonviral vectors to replace a missing or defective gene in order to correct a disease ...

Adeno-associated virus (AAV) mediated gene transfer (GT) is a rapidly emerging therapeutic approach for targeted gene delivery for the amelioration of inherited diseases, with 8 approved in-vivo AAV gene replacement therapies across the United States, Canada, and …