ARTICLE | Distillery Therapeutics

Endocrine / Metabolic

November 14, 2017 10:59 PM UTC

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...

BCIQ Company Profiles

University at Buffalo

BCIQ Target Profiles

Acid alpha glucosidase (GAA)