Claims for Patent: 10,961,522

Summary for Patent: 10,961,522

Title:	Highly potent acid alpha-glucosidase with enhanced carbohydrates
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.
Inventor(s):	Gotschall Russell, Do Hung V.
Assignee:	Amicus Therapeutics, Inc.
Application Number:	US16252505
Patent Claims:	2. The method of claim 1 , wherein the rhGAA comprises 3.0 to 4.0 mol M6P per mol rhGAA.3. The method of claim 1 , wherein the rhGAA comprises 4.0 to 5.0 mol M6P per mol rhGAA.4. The method of claim 1 , wherein about 45%-55% of the N-glycans on the rhGAA are complex type N-glycans.5. The method of claim 1 , wherein 50% of the N-glycans on the rhGAA are complex type N-glycans.6. The method of claim 1 , wherein the rhGAA comprises at least one bis-phosphorylated N-glycan per rhGAA.7. The method of claim 1 , wherein the rhGAA further comprises(a) 2.0 to 8.0 mol sialic acid per mol rhGAA; or(b) at least 4 mol sialic acid per mol rhGAA.8. The method of claim 1 , wherein the method further comprises administering a pharmacological chaperone claim 1 , and wherein said composition and said pharmacological chaperone are either administered as a single pharmaceutical composition or administered separately.9. The method of claim 8 , wherein the pharmacological chaperone is N-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof.10. The method of claim 1 , wherein the rhGAA is conjugated to a targeting moiety.11. The method of claim 1 , wherein the method modulates lysosomal proliferation.12. The method of claim 1 , wherein the method modulates autophagy.