IP Library › Granted Patent US 11,591,583
Granted Patent B2
US 11,591,583 · App. 17/665,179 · Granted Feb 28, 2023

Highly potent acid alpha-glucosidase with enhanced carbohydrates

Inventors: Russell Gotschall (Doylestown, PA); Hung V. Do (New Hope, PA)
Assignee: Amicus Therapeutics, Inc.
C12N9/2465A61K48/005C12N15/09C12Y302/0102C12N2510/02
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Quick Facts
Patent No.
US 11,591,583
App. No.
17/665,179
Granted
Feb 28, 2023
Kind
B2
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.

Claims (16)

1. A composition comprising recombinant human alpha glucosidase (rhGAA), wherein 40%-60% of the N-glycans on the rhGAA are complex type N-glycans, and wherein each rhGAA molecule on average comprises at least one bis-phosphorylated mannose-6-phosphate (bis-M6P glycan).

2. The composition of claim 1 , wherein at least 17% of the total glycans on the rhGAA are bis-M6P glycans.

3. The composition of claim 1 , wherein the rhGAA comprises the amino acid sequence that is at least 90% identical to SEQ ID NO: 4.

4. The composition of claim 1 , wherein the rhGAA comprises at least 3.0 mol mannose-6-phosphate (M6P) residues per mol rhGAA.

5. The composition of claim 4 , wherein the rhGAA comprises at least 4.0 mol M6P residues per mol rhGAA.

6. The composition of claim 4 , wherein the rhGAA comprises at least 5.0 mol M6P residues per mol rhGAA.

7. The composition of claim 4 , wherein the rhGAA comprises at least 6.0 mol M6P residues per mol rhGAA.

8. The composition of claim 4 , wherein the rhGAA comprises at least 7.0 mol M6P residues per mol rhGAA.

9. The composition of claim 4 , wherein the rhGAA comprises from 3.0 mol to 6.0 mol M6P residues per mol rhGAA.

10. The composition of claim 4 , wherein the rhGAA comprises from 3.0 mol to 7.0 mol M6P residues per mol rhGAA.

11. A pharmaceutical composition comprising the rhGAA of claim 1 and at least one pharmaceutically acceptable carrier or excipient.

12. The pharmaceutical composition of claim 11 , wherein the pharmaceutically acceptable carrier is water.

13. The pharmaceutical composition of claim 11 , wherein the composition is in the form of a lyophilized powder.

14. The pharmaceutical composition of claim 11 , wherein the composition comprises mannitol.

15. The pharmaceutical composition of claim 11 , wherein the composition comprises polysorbate 80.

16. The pharmaceutical composition of claim 11 , wherein the composition comprises a pH buffer.

Assignments (3)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →
Cited By (2)
US 12,428,487 US 12,662,545