AAV manufacturing for clinical use: Insights on current challenges from the upstream process
AAV manufacturing for clinical use: Insights on current challenges from the upstream process
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DOI：
10.1016/j.cobme.2021.100353
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发表时间：
2021-11-06
影响因子：
3.9
通讯作者：
Suh, Junghae
中科院分区：
文献类型：
--
作者：
Dobrowsky, Terrence;Gianni, Davide;Suh, Junghae
随着基因治疗正在经历第二波关注，重组腺相关病毒（rAAV）因其良好的安全性和长期转基因表达而迅速成为最具吸引力的病毒转移工具之一。最近批准的 rAAV 分子用于治疗先天性黑蒙和脊髓性肌萎缩症，为开发几种基于 rAAV 的治疗药物（针对不断增加的适应症）铺平了道路。为了支持这种快速增长，该领域迫切需要稳健、可扩展且经济可行的制造工艺。本次综述将概述目前 rAAV 生产制造层面采用的最常见平台，重点关注上游流程中遇到的挑战和瓶颈。
As gene therapy is experiencing a second wave of interest, recombinant adeno-associated virus (rAAV) has rapidly emerged as one of the most attractive viral transfer tools, thanks to its favorable safety profile and long-term transgene expression. The recent approval of rAAV molecules for the treatment of congenital amaurosis and spinal muscular atrophy has been paving the road for the development of several rAAVbased therapeutics targeting a continuously increasing range of indications. To support this rapid growth, the field is in high demand for robust, scalable and economically viable manufacturing processes. This review will provide an overview of the most common platforms currently adopted at the manufacturing level for rAAV production, focusing on the challenges and bottlenecks experienced in the upstream process.