M103c - Regulator’s Perspective on AAV

Scientific literature shows that, recombinant AAV vectors derived from human- versus insect cell manufacturing platforms exhibit significant different potency. The causes for these differences seem currently not entirely clear. It is reasonable to conclude that we do not yet know certain important quality attributes of the rAAV products. This lack of knowledge is especially critical in case of manufacturing changes and scale-up activities during development of the manufacturing process because comparability of the resulting rAAV material has to be ensured. Thus, with respect to a future Marketing Authorization Application it is important to apply a state-of-the-art characterization panel in order to have the best possible picture of the manufactured rAAV material.