Solving the AAV scalability challenge

Posted: 5 December 2024

Gene to GMP AAV manufacturing partner, Ascend, made a presentation at ESACT about leveraging technology to build a flexible and reliable platform. Working with Sartorius, the team has demonstrated consistent productivity (viral genome and capsid titers), quality (packaged host-cell and plasmid DNA, percent full capsids), and potency from 15 ml to 200 L scales with both the proprietary split two plasmid EpyQ™ AAV production system or traditional triple transfection. A review of the challenges and results follows.

Introduction

Adeno-associated viral (AAV) vectors have a clearly demonstrated ability to successfully deliver gene therapies. Minimal immunogenicity and significantly limited ability to replicate offer important advantages, as does the varied tropism of an ever-expanding array of AAV serotypes …