Andelyn Capacity Update April 2025: Cell & Gene Therapy

Adeno-associated viruses (AAVs) are widely recognized as promising vectors for gene therapy due to their safety profile, low immunogenicity, and ability to transduce a variety of cell types. However, the successful implementation of AAV-based therapies on a commercial scale remains a significant challenge, particularly in terms of scalable production and purification processes. Successful scale-up and scale-down models are crucial to the commercialization of AAV gene therapies, enabling cost-effective and large-scale manufacturing that meets regulatory standards while delivering therapeutic efficacy.

Where Scale-up is a matter of engineering, designed to transition laboratory-scale production systems to industrial-scale facilities while maintaining high efficiency and reproducibility. Conversely, scale-down models serve as cost-effective and time-efficient tools to demonstrate process understanding and control as well as predict and troubleshoot issues that may arise during large-scale production.

Here we will discuss how proper optimization leads to success in both scale-up and scale-down models for AAV production by focusing on critical parameters such as cell culture conditions, viral vector yield, and downstream purification strategies.