Gene therapy: challenges in cell culture scale-up
Jenny Shupe, An Zhang, Daniel C. Odenwelder, Terrence M. Dobrowsky
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.
Topics & Concepts
BottleneckGenetic enhancementScale (ratio)BiotechnologyProduct (mathematics)ModalitiesProcess (computing)Production (economics)Computer scienceComputational biologyGeneBiologyGeneticsEconomicsSocial sciencePhysicsMathematicsGeometryOperating systemQuantum mechanicsMacroeconomicsSociologyEmbedded systemVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in InsectsCAR-T cell therapy research