Abeona Therapeutics evaluates a scalable suspension-based platform for production of viral vector for MPS-IIIA
Cell & Gene Therapy Insights 2022; 8(1), 143
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.