Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical <i>Ex Vivo</i> Gene Therapy Application

Abstract excerpt

From the perspective of a pilot clinical gene therapy trial for Wiskott–Aldrich syndrome (WAS), we implemented a process to produce a lentiviral vector under good manufacturing practices (GMP). The process is based on the transient transfection of 293T cells in Cell Factory stacks, scaled up to harvest 50 liters of viral stock per batch, followed by purification of the vesicular stomatitis virus...

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