Attributed to their safety profiles and transduction efficiencies, recombinant Adeno-associated virus (AAV) vectors are widely used for treatment of human genetic diseases. However, the high cost and low productivity in AAV vector manufacturing process limit the clinical and commercial development of these life-changing therapies for more patients. Recently, we reported the development of high-performing, HEK293 cell based, helper virus-free AAV stable producer cell lines (PCL) that significantly increased the AAV productivity (> 1E12 vg/mL at harvest) compared to transient transfection-based process.
In this new case study, we describe the generation of high-titer stable PCL for AAV vectors using a clinically relevant transgene and an engineered capsid provided by our partner. The beta test results confirmed the robust AAV PCL development process from stable pools to selected single cell clones, which produced significantly higher titers than the transient transfection process. Therefore, our Xcite® AAV stable PCL represents a next-generation technology to enable large-scale AAV manufacturing at markedly reduced cost and complexity.