Developing an Intensified AAV Production Process to Reduce CoGs

There remains a significant need to improve adeno-associated virus (AAV) manufacturing platforms to achieve robust, high-yielding, scalable and cost-efficient processes. At Ultragenyx, we employ a producer cell line – helper-virus based infection process for AAV production which is reproducibly scalable to 2000L. To further improve on our batch process platform, we have developed a modified batch process using perfusion for AAV production to increase cell density to achieve high volumetric AAV yield. The upstream process demonstrated AAV productivity to ≥5E11 GC/mL at 250L scale, enabling delivery of an affordable AAV product suitable for clinical use.

Presented by, Dr. Wei Xue, Associate Director at Ultragenyx Pharmaceuticals

Dr. Wei Xue is Associate Director at Ultragenyx Pharmaceuticals, focusing on early-stage upstream process development and IND-enabling activities on recombinant adeno-associated virus (rAAV) based gene therapy. Before Ultragenyx, he worked at Regeneron Pharmaceuticals as a Sr. Process Scientist for 5 years, leading efforts on process understanding, deviation investigation and raw material identification activities to support Upstream Manufacturing. He earned his Ph.D in Chemical Engineering at the Ohio State University in 2016.