[HTML][HTML] Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

B Bertin, P Veron, C Leborgne, JY Deschamps… - Scientific reports, 2020 - nature.com
B Bertin, P Veron, C Leborgne, JY Deschamps, S Moullec, Y Fromes, F Collaud, S Boutin…
Scientific reports, 2020nature.com
Neutralizing antibodies directed against adeno-associated virus (AAV) are commonly found
in humans. In seropositive subjects, vector administration is not feasible as antibodies
neutralize AAV vectors even at low titers. Consequently, a relatively large proportion of
humans is excluded from enrollment in clinical trials and, similarly, vector redosing is not
feasible because of development of high-titer antibodies following AAV vector
administration. Plasmapheresis has been proposed as strategy to remove anti-AAV …
Abstract
Neutralizing antibodies directed against adeno-associated virus (AAV) are commonly found in humans. In seropositive subjects, vector administration is not feasible as antibodies neutralize AAV vectors even at low titers. Consequently, a relatively large proportion of humans is excluded from enrollment in clinical trials and, similarly, vector redosing is not feasible because of development of high-titer antibodies following AAV vector administration. Plasmapheresis has been proposed as strategy to remove anti-AAV antibodies from the bloodstream. Although safe and relatively effective, the technology has some limitations mainly related to the nonspecific removal of all circulating IgG. Here we developed an AAV-specific plasmapheresis column which was shown to efficiently and selectively deplete anti-AAV antibodies without depleting the total immunoglobulin pool from plasma. We showed the nearly complete removal of anti-AAV antibodies from high titer purified human IgG pools and plasma samples, decreasing titers to levels that allow AAV vector administration in mice. These results provide proof-of-concept of a method for the AAV-specific depletion of neutralizing antibodies in the setting of in vivo gene transfer.
nature.com