Indeed, recombinant Adeno-Associated Virus (AAV) are viral vectors of choice for in vivo gene therapy with already two products currently approved in the market. However, the manufacturing process to produce large amounts of AAV vectors remains a major challenge in particular with the increasing clinical and market demands, and the necessity of systemic administration of high doses to achieve therapeutic efficiency. Our translational vector core (CPV) project aims at addressing these challenges from fundamental “vectorology” to preindustrial manufacturing.
The translational vector core (CPV) has expertise in large scale rAAV vectors production. During 25 years CPV has developed rAAV production platforms based on transient transfection of HEK293 adherent cells or recently in suspension cells and also based on suspension insect cells infected with recombinant baculovirus. The improvement of rAAV processes cannot be dissociated from the characterization of these vectors. Today a large number of technologies are being developed to deep to characterize rAAV more precisely in order to increase the therapeutic effect with less immunogenicity.