Viral vectorology for gene therapy
July 16-20, 2017
Given the recent successes of in vivo gene therapy (for monogenetic diseases or oncolytic therapy), as well as the expanding search of ex-vivo gene therapy for cancer immunotherapy (ex, CAR-T), viral vectorology will need added R&D efforts for the next decade. As for mAbs, in the early nineties, the titers and quality of viruses as vectors are poor and thus costly to produce, requiring much improvement to substantially reduce costs of goods (COGs), similarly to what took place over the last two and a half decades, when mAb titers were improved over 70 times, through continuous process development. As for other areas of biotechnology, training is needed in basic understanding of molecular biology of the cell – virus interaction, analyticals and then upstream processes (in particular continuous – perfusion, as some viruses are highly labile) coupled with new ways of extraction and purification. Partnerships with companies developing the new tools, in particular based on single use options, while process development is carried out for early stage biotechnology companies, has been one of the back bones of iBET operation over the last twenty years. Thus, a discussion will be conducted on how “learning by doing” in R&D programmes, Ph.D.’s but also M.Sc., is critical to train those that will, hopefully, do for viral vectorology what took place recently for mAbs.
Paula Alves, "Viral vectorology for gene therapy" in "Biochemical and Molecular Engineering XX", Wilfred Chen, University of Delaware, USA Nicole Borth, Universität für Bodenkultur, Vienna, Austria Stefanos Grammatikos, UCB Pharma, Belgium Eds, ECI Symposium Series, (2017). http://dc.engconfintl.org/biochem_xx/119