Gene therapy: challenges in cell culture scale-up

Jenny Shupe; An Zhang; Daniel C Odenwelder; Terrence Dobrowsky

doi:10.1016/j.copbio.2022.102721

Gene therapy: challenges in cell culture scale-up

Curr Opin Biotechnol. 2022 Jun:75:102721. doi: 10.1016/j.copbio.2022.102721. Epub 2022 Apr 7.

Authors

Jenny Shupe¹, An Zhang², Daniel C Odenwelder², Terrence Dobrowsky²

Affiliations

¹ Pharmaceutical Operations and Technology, Biogen, Inc., 225 Binney Street, Cambridge, MA 02142, USA. Electronic address: jenny.shupe@biogen.com.
² Pharmaceutical Operations and Technology, Biogen, Inc., 225 Binney Street, Cambridge, MA 02142, USA.

PMID: 35398708
DOI: 10.1016/j.copbio.2022.102721

Abstract

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.

Publication types

Review

MeSH terms

Cell Culture Techniques
Dependovirus* / genetics
Gene Transfer Techniques
Genetic Therapy / methods
Genetic Vectors* / genetics