Adeno-associated virus (AAV) is being developed for gene therapy applications. This virus type presents several advantages over alternate vectors for therapeutic gene delivery. AAV is not considered pathogenic and transduces stably dividing and non-dividing cells. AAV also shows good serotype specificity to various cell types for targeted gene delivery.
The present invention describes a highly scalable adeno-associated virus (AAV) vector production method in insect cells. The system for producing recombinant AAV (rAAV) uses the AAV Rep protein and an AAV ITR. This production method produces virus particles much more efficiently than the standard mammalian cell culture system. Yields of rAAV produced in Sf9 cells exceed 10e15 per liter for some constructs. The improvement in production efficiency translates into lower production costs and potential for commercial scale manufacturing. In addition, all serotypes of AAV can be produced, with the respective AAV serotype vectors available for the immediate scale up of AAV production.
This technology will give a company producing large quantities of AAV a significant competitive advantage over traditional AAV production methods.