Endocrine / Metabolic
Mouse studies suggest GAA-loaded nanoparticles could help treat Pompe’s disease without generating anti-GAA antibodies associated with resistance to enzyme replacement therapy. The nanoparticles consist of low-immunogenic anionic phosphatidylinositol liposomes loaded with GAA. In a mouse model of Pompe’s disease, the GAA-loaded nanoparticles decreased serum anti-GAA antibody titers and the disease markers of glycogen levels in the heart and triceps muscle compared with free GAA. Next steps could include testing the long-term efficacy and safety of the GAA-loaded nanoparticles the model
Amicus Therapeutics Inc. has ATB200, an enzyme replacement therapy (ERT) consisting of recombinant human GAA (rhGAA), in Phase I/II testing to treat Pompe’s disease. Amicus also has ATB200/AT2221, a co-administered therapy comprising ATB200, a rhGAA, and the pharmacological chaperone AT2221, in Phase I/II testing to treat Pompe’s disease...
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