Bioproduction of gene therapy medicines : use cases

This activity explores the production of non-viral vectors for tumor treatment, from the selection of the therapeutic strategy to the manufacturing of the drug substance and the specific quality controls of mRNA based products.

This activity presents the steps of viral vector bioproduction for the treatment of Parkinson’s disease, from in vitro transcription to quality control of the drug substance.

This activity addresses autologous ex vivo gene therapy for the treatment of sickle cell disease, identifying the specific quality controls for cell-based gene therapies.