Gene therapy: challenges in cell culture scale-up

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.

Automated summary

Gene therapy aims to cure diseases caused by genetic defects, and rAAV is currently the preferred tool for therapeutic gene delivery. However, manufacturing enough rAAV products remains a bottleneck in the industry. In the past decade, various production platforms have been developed with improvements in productivity, but limitations still exist.