Scalable Purification of Viral Vectors for Gene Therapy: An Appraisal of Downstream Processing Approaches

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Figure 1: Viral vector production

Figure 1: Viral vector production

Gene therapy is the transfer of genetic material to a patient’s cells to achieve a therapeutic effect. Therapeutic DNA is largely delivered using viral vector systems based on adenoviruses (Ad), adenoassociated viruses (AAV), and lentiviruses (LV). With the application of such viral vectors as clinical therapeutics growing, scalable commercial processes (particularly for purification) are being investigated and optimized to best ensure that critical quality attributes (CQAs) are retained. Herein we review viral vector purification techniques and the effect of different characteristics of vector classes on the selection of optimal unit operations.

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Maya Fuerstenau-Sharp is a field-marketing manager at Sartorius Stedim (Göttingen, Germany). David Pettitt is an academic surgeon, CASMI research associate, associate at IP Asset Ventures, Ltd, and a doctoral researcher at the University of Oxford. Kim Bure is director of regenerative medicine at Sartorius Stedim. James Smith is a CASMI research associate and doctoral researcher in the University of Oxford’s Nuffield Department of Orthopedics, Rheumatology, and Musculoskeletal Sciences, and an associate at IP Asset Ventures, Ltd. Jamie Ware is a CASMI research associate. Corresponding author David Brindley is founder and academic director of CASMI’s Translational Stem Cell Consortium, senior healthcare translation research fellow in the medical sciences division’s department of paediatrics at the University of Oxford, Cooksey-Saïd fellow in healthcare translation at the University of Oxford’s Saïd Business School, honorary senior research associate at the University College London School of Pharmacy’s Centre for Behavioral Medicine, a research fellow in cell therapy commercialization at the Harvard Stem Cell Institute, and regenerative medicine regulation and risk management lead at the USCF Stanford Center of Excellence in Regulatory Science and Innovation (CERSI) in Stanford, CA.