IP Library Granted Patent US 10,258,671
Granted Patent B2
US 10,258,671 · App. 15/830,655 · Granted Apr 16, 2019

Compositions and methods for treating platelet storage pool deficiency, gray platelet syndrome and quebec platelet disorder

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Quick Facts
Patent No.
US 10,258,671
App. No.
15/830,655
Granted
Apr 16, 2019
Kind
B2
Abstract

The present invention relates to compositions and methods for treating diseases of coagulation using a NPP4 polypeptide.

Claims (22)

1. A method of treating Platelet Storage Pool Deficiency in a human subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of an isolated soluble NPP4 polypeptide comprising amino acid residues 1-407 of SEQ ID NO: 1.

2. The method of claim 1 , wherein the isolated soluble NPP4 polypeptide is a recombinant NPP4 polypeptide.

3. The method of claim 1 , wherein the isolated soluble NPP4 polypeptide lacks an NPP4 transmembrane domain.

4. The method of claim 1 , wherein the isolated soluble NPP4 polypeptide consists of amino acid residues 1-407 of SEQ ID NO: 1.

5. The method of claim 1 , wherein the isolated soluble NPP4 polypeptide is administered in combination with at least one other agent useful in treating bleeding.

6. The method of claim 1 , wherein the isolated soluble NPP4 polypeptide is administered acutely or chronically.

7. The method of claim 1 , wherein the isolated soluble NPP4 polypeptide is administered locally, regionally or systemically.

8. The method of claim 1 , wherein the Platelet Storage Pool Deficiency is a Delta storage pool deficiency.

9. A method of treating Quebec Platelet disorder in a human subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of an isolated soluble NPP4 polypeptide comprising amino acid residues 1-407 of SEQ ID NO: 1.

10. The method of claim 9 , wherein the isolated soluble NPP4 polypeptide is a recombinant NPP4 polypeptide.

11. The method of claim 9 , wherein the isolated soluble NPP4 polypeptide lacks an NPP4 transmembrane domain.

12. The method of claim 9 , wherein the isolated soluble NPP4 polypeptide consists of amino acid residues 1-407 of SEQ ID NO: 1.

13. The method of claim 9 , wherein the isolated soluble NPP4 polypeptide is administered in combination with at least one other agent useful in treating bleeding.

14. The method of claim 9 , wherein the isolated soluble NPP4 polypeptide is administered acutely or chronically.

15. The method of claim 9 , wherein the isolated soluble NPP4 polypeptide is administered locally, regionally or systemically.

16. A method of treating Gray Platelet Syndrome in a human subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of an isolated soluble NPP4 polypeptide comprising amino acid residues 1-407 of SEQ ID NO: 1.

17. The method of claim 16 , wherein the isolated soluble NPP4 polypeptide is a recombinant NPP4 polypeptide.

18. The method of claim 16 , wherein the isolated soluble NPP4 polypeptide lacks an NPP4 transmembrane domain.

19. The method of claim 16 , wherein the isolated soluble NPP4 polypeptide consists of amino acid residues 1-407 of SEQ ID NO: 1.

20. The method of claim 16 , wherein the isolated soluble NPP4 polypeptide is administered in combination with at least one other agent useful in treating bleeding.

21. The method of claim 16 , wherein the isolated soluble NPP4 polypeptide is administered acutely or chronically.

22. The method of claim 16 , wherein the isolated soluble NPP4 polypeptide is administered locally, regionally or systemically.

Assignments (1)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →