Claims for Patent: 10,208,299

Summary for Patent: 10,208,299

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:	US15515808
Patent Claims:	2. The composition of claim 1 , wherein the rhGAA comprises 3.0 to 4.0 mol M6P per mol rhGAA.3. The composition of claim 1 , wherein the rhGAA comprises 4.0 to 5.0 mol M6P per mol rhGAA.4. The composition of claim 1 , wherein the rhGAA further comprises at least 4 mol sialic acid per mol rhGAA.5. The composition of claim 1 , wherein the rhGAA further comprises 2.0 to 8.0 mol sialic acid residues per mol of rhGAA.6. The composition of claim 1 , wherein the composition further comprises a pharmacological chaperone.7. The composition of claim 1 , wherein the rhGAA comprises at least one bis-phosphorylated N-glycan per rhGAA.8. The composition of claim 1 , wherein about 45%-55% of the N-glycans on the rhGAA are complex type N-glycans.9. The composition of claim 1 , wherein 50% of the N-glycans on the rhGAA are complex type N-glycans.10. The composition of claim 1 , wherein the rhGAA comprises 4 mol M6P per mol rhGAA claim 1 , 4.5 mol sialic acid per mol rhGAA claim 1 , and at least one bis-phosphorylated N-glycan per rhGAA.11. The composition of claim 10 , wherein the composition further comprises the pharmacological chaperone N-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof.12. A method for treating Pompe disease in a subject in need thereof claim 1 , the method comprising administering the composition of to the subject in an amount sufficient to treat Pompe disease.13. The method of claim 12 , wherein said composition is administered to cardiac muscle of the subject.14. The method of claim 12 , wherein said composition is administered to quadriceps claim 12 , triceps claim 12 , or other skeletal muscle of the subject.15. The method of claim 12 , wherein said composition is administered to the diaphragm of the subject.16. The method of claim 12 , wherein said composition is combined with a pharmacological chaperone claim 12 , and wherein said composition and said pharmacological chaperone are either administered as a single pharmaceutical composition or administered separately.17. The method of claim 12 , wherein said composition is combined with 1-deoxynojirimycin or a pharmaceutically acceptable salt thereof claim 12 , and wherein said composition and said 1-deoxynojirimycin or pharmaceutically acceptable salt thereof are either administered as a single pharmaceutical composition or administered separately.18. The method of claim 12 , wherein said composition is combined with N-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof claim 12 , and wherein said composition and said N-butyl-deoxynojirimycin or pharmaceutically acceptable salt thereof are either administered as a single pharmaceutical composition or administered separately.