Solving The AAV Scalability Challenge

By Ahmed Youssef, Senior Manager of Upstream Process Development at Ascend; and Melanie Plath, Application Specialist Bioreactor Technologies at Sartorius Stedim Biotech GmbH

Adeno-associated viral (AAV) vectors have proven to be powerful tools for delivering gene therapies due to their minimal immunogenicity, non-replicating nature, and versatile tropism enabled by an expanding array of engineered serotypes. These characteristics make AAV an ideal candidate for a wide range of therapeutic applications. However, despite their promise, the pharmaceutical industry is still in the early stages of fully realizing AAV’s potential. Developing cost-effective, seamlessly scalable, and standardized manufacturing processes remains a critical challenge.

Recognizing these needs, Ascend Advanced Therapies has developed a flexible and enabling AAV manufacturing platform to support the industry's growth. Central to this platform is a well-characterized suite of lab- to production-scale bioreactors from Sartorius Stedim Biotech (Sartorius), capable of scaling from 250 mL to 200 L. Positive results have been achieved using both the EpyQ AAV production system and customer-specific triple transfection processes, demonstrating robustness and scalability for commercial production needs.