I’m thrilled to share the first results from our large-scale AAV production using the AAVone system in the XDR200 bioreactor. Utilizing the pAAVone-AAV9-CMV-EGFP plasmid, we achieved approximately 1e12 VG/L of AAV9 in VPC2.0 cells—even under non-optimized conditions. Notably, the full particle percentage in crude samples reached 28.5%.

The AAVone system demonstrates impressive efficiency, with a high recovery rate of 69% and 6.55e14 VG/L of purified AAV9. This was achieved using a remarkably low DNA input of just 0.3 µg per 1 million cells.

More excitingly, with optimized transfection reagents, media, and enhancers, we’ve observed a 3-fold increase in productivity in 125 mL flasks, reaching over 5e15 VG/L in crude titers. We are confident these optimizations can be scaled to bioreactors, with the potential to exceed 2e15 VG/L of purified AAV9.

At this yield, just one liter of culture could be sufficient to treat one patient, significantly driving down the cost of AAV gene therapy.