High Yield

Through cell line development and process development, our production platform reaches the high level of production yield among those reported in the AAV industry. The high yield leads to low manufacturing cost, and competitive drug pricing to benefit patients and payors across the world. 

High Purity

Our production platform produces high percentage of full AAV vectors containing the therapeutic DNA, and thus reduces the generation of empty capsids. In addition, our platform produces AAVs with low DNA impurities and low protein impurities. The high level of empty capsids, and/or the high impurity DNA or proteins, in an AAV drug are likely associated with reported Serious Adverse Event (SAE) in various AAV clinical trials. With the high AAV purity, we improve the drug safety profile.

Scalability

Our process is reproducible at different scales from 2L to 500L in single-use bioreactors. The lower levels of empty capsids produced in our upstream process enables all downstream purification steps to be scalable and removes bottlenecks (e.g. ultracentrifugation) present in other production systems. The good scalability shortens the development time from research to manufacturing, and removes uncertainty of the AAV productivity in the program planning and execution.