Effect of AT2220 on ERT-Related Immunogenicity Measured ex vivoBy stabilizing the folded and active form of the rhGAA enzyme, AT2220 may mitigate ERT-induced immunogenicity since unfolded and aggregated proteins are generally more antigenic than properly folded proteins. Recent published studies show that approximately 40% of the administered ERT can be captured by circulating antibodies and infusion associated reactions occur in approximately 50% of Pompe patients receiving ERT infusions. 2 Initial ex vivo studies using T cells derived from blood from 50 healthy donors demonstrated that the addition of AT2220 may significantly reduce the immunogenicity of Myozyme and Lumizyme. The studies utilized Antitope Ltd.'s EpiScreen™ assay and are being repeated in samples from the Pompe patients in Study 010. Results from these ex vivo studies may help to guide the clinical investigation of the effects of AT2220 on ERT-related immunogenicity.
Amicus Therapeutics Announces Positive Results From All Four Cohorts In Phase 2 Chaperone-Enzyme Replacement Therapy (ERT) Co-Administration Study For Pompe Disease
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