Live30 webinars are thirty minute presentations designed to update you on the latest innovations, applications and data in a fast yet interactive format.
Viral vector-based gene therapies are bringing life changing treatments to many patients and families. One investigational viral vector-based gene therapy in development is for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA). This gene replacement therapy uses an AAV capsid to deliver a functional hSGSH gene.
If approved, a scalable upstream production process is needed to provide the necessary manufacturing capacity for this life-changing therapy to reach patients suffering from this disease. In this webinar, new insights into scaling up production of high-titer and high-quality viral vectors at clinical and commercial scales are presented.
Participants will learn: