Gene therapy: challenges in cell culture scale-up

被引:16
作者
Shupe, Jenny [1 ]
Zhang, An [1 ]
Odenwelder, Daniel C. [1 ]
Dobrowsky, Terrence [1 ]
机构
[1] Biogen Inc, Pharmaceut Operat & Technol, 225 Binney St, Cambridge, MA 02142 USA
关键词
ADENOASSOCIATED VIRAL VECTORS; SERUM-FREE PRODUCTION; TRANSIENT TRANSFECTION; SUSPENSION CELLS; AAV VECTOR; HIGH-TITER; VIRUS; BIOREACTOR; PARTICLES; PLATFORM;
D O I
10.1016/j.copbio.2022.102721
中图分类号
Q5 [生物化学];
学科分类号
071010 ; 081704 ;
摘要
Gene therapy is designed to cure various diseases resulting from genetic defects. Currently, recombinant adeno-associated viral vectors (rAAV) are the vehicles of choice for therapeutic gene delivery in vivo. To date, manufacturing sufficient rAAV product to meet rapidly expanding clinical demand remains a bottleneck in the industry. In the past decade, multiple production platforms have been rapidly implemented with encouraging improvements in productivity and scalability. In this review, we discuss the advantages and limitations of the most popular production platforms in the industry with a focus on the cell culture process scale-up.
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页数:5
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