Highly potent acid alpha-glucosidase with enhanced carbohydrates
AssigneeAmicus Therapeutics, Inc.
InventorsRussell Gotschall, Hung V. Do
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Abstract
Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.
Claims13
-27. (canceled)
A method for metabolizing, degrading, removing, or otherwise decreasing glycogen in tissue, muscle, muscle fiber, muscle cells, lysosomes, organelles, cellular compartments, or cytoplasm of a subject, comprising administering to the subject a composition comprising recombinant human acid alpha-glucosidase (rhGAA) produced from Chinese hamster ovary (CHO) cells, wherein 40%-60% of the N-glycans on the rhGAA are complex type N-glycans and the rhGAA comprises 3.0-5.0 mol mannose-6-phosphate (M6P) per mol rhGAA.
Dependencies are inferred from the claim text.