In the past few years, the resurgence of cell-based immunotherapies — and, by extension, gene therapies — has accelerated as products move rapidly from academic research laboratories into commercial development. A successful clinical trial by St. Jude Children’s Research and the University College London of a gene therapy for hemophilia B was a seminal translational event (1), as was the licensing of the University of Pennsylvania’s gene-modified chimeric antigen receptor T-cell technology (CAR-T) for leukemia by Novartis (2). CAR-T cells and gene therapies are now among the most dynamic areas for biotechnology investment and development. Both gene therapies and gene-modified cell therapies require large amounts of viral vectors, either for direct delivery to patients or for ex vivo modification of cells that are later dosed to patients.
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