Overcoming upstream & downstream process barriers for large-scale AAV production
Cell & Gene Therapy Insights 2023; 9(7), 1025–1036
Given the broad treatment potential and demonstrated efficacy of recently approved viral vector-based gene therapies, there is an established need for reproducible and consistent manufacturing solutions that yield high titer, high quality viral particles for clinical applications. It is critical to implement a robust manufacturing process that addresses the upstream and downstream production challenges of obtaining sufficient titers and purity for in vivo applications, whilst also meeting safety and regulatory requirements for clinical use.This article discusses key barriers to addressing clinical needs and market supply of gene therapies. We will explore the implementation of solutions to overcome these issues, thereby optimizing AAV manufacturing processes and accelerating the development of safe and effective gene therapies.