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April 15-16, 2019 | Milan, Italy
OF EXCELLENCE
IN INTERNATIONAL
MEETINGS
alliedacademies.comYEARS
Joint Event on
&
PUBLIC HEALTH,
EPIDEMIOLOGY AND NUTRITION
2
nd
World Congress on
CELL AND GENE THERAPY
2
nd
International Conference on
Cell and Gene Therapy 2019 & Public Health Congress 2019
Archives of General Internal Medicine | ISSN: 2591-7951 | Volume 3
DEVELOPMENT OF A SCALABLE PRODUCTION PLATFORM FOR RECOMBINANT
ADENO-ASSOCIATED VIRUS VECTORS IN SUSPENSION HEK293 CELLS
Lawrence Tam
Oxford Genetics Ltd., United Kingdom
R
ecombinant Adeno Associated Viral vectors (rAAV) have emerged as a powerful tool for
in vivo
gene deliv-
ery owing to their unique characteristics, including a high safety profile and ability to drive long-term gene
expression in humans. Current successes in AAV-mediated clinical trials have prompted pharmaceutical com-
panies to quickly move into the AAV gene therapy market and resulted in growing demand for clinical-grade
rAAV vectors. In order to address these requirements, Oxford Genetics has developed a scalable manufacturing
platform based on optimized expression plasmids and a clonal cGMP-compliant suspension HEK293 cell line.
While this platform facilitates representative material generation for early stage clinical trials, it also allows the
transition to packaging and producer cell lines based on the same underlying genetic components, cell line
background and process/media system as vector requirements increase. Stable cell lines enable the develop-
ment of rapid, robust and high-productivity processes, with reduced cost of goods and simplified supply chain.
Here we present an update on characterization of these cell lines and internal process development activities
to demonstrate associated product quality assurance and scale-up.
Arch Gen Intern Med 2019, Volume 3 | DOI: 10.4066/2591-7951-C2-027