Transforming Gene Therapy With A Scalable Platform Solution For AAV Production

By Mikkal Blick, Monika Szeszel, Frank Fabian, Jaime Garcia Escalante, Mitchell Simmonds, Phillip Jones, Yu Zhou, Ayan Dey, Hugh Murray, Eva Fong, and Michael Shen, Viral Vector Process Development, Millipore® CTDMO Services, Carlsbad, CA, USA.

Gene therapies offer new possibilities for treating rare and complex diseases, but producing the viral vectors used to deliver therapeutic genes can be costly and time-intensive. A platform approach to adeno-associated virus (AAV) development can simplify this process by optimizing manufacturing elements that remain consistent across different therapeutic genes. Reusing these established parameters reduces the need to build each production process from the ground up, helping development teams control costs and move promising therapies toward the clinic faster.

Examine how a robust AAV manufacturing platform achieves high product yields while supporting scale-up to 1,000 L bioreactors, providing a practical foundation for efficient and scalable clinical material production.