Abstract
Viral vector-based gene therapies are bringing life-changing treatments to patients. One investigational therapy in development is a novel hSGSH gene replacement therapy for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA), which uses an AAV capsid to deliver a functional gene. If this therapy is approved, a scalable upstream production process is needed to provide the necessary manufacturing capacity to allow it to reach patients. In a recent webinar, new insights into scaling up production of high titer and high-quality viral vectors at clinical and commercial scales were discussed. Some key experimental results are highlighted here.
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