GAA and hemophilia: We have demonstrated the ability of NPs encapsulating rapamycin to inhibit the formation of ADAs against a variety of biologic drugs in preclinical studies, including coagulation factor VIII in hemophilia A mice (19), myozyme (or acid alpha glucosidase) in a murine model of Pompe disease (17), humira in a spontaneous model of inflammatory arthritis, and pegylated uricase enzyme in both Urox-deficient mice and non-human primates (16).