Reagents and methods for screening MPS I, II, IIIA, IIIB, IVA, VI, and VII

Patent metadata

Field	Value
Publication number	US-11618764-B2
Application number	US-201816224001-A
Country	US
Kind code	B2
Filing date	Dec 18, 2018
Priority date	Sep 5, 2013
Publication date	Apr 4, 2023
Grant date	Apr 4, 2023

Abstract

Reagents, methods, and kits for assaying enzymes associated with lysosomal storage diseases MPS-I, MPS-II, MPS-IIIA, MPS-IIIB, MPS-IVA, MPS-VI, and MPS VII.

First claim

The embodiments of the invention in which an exclusive property or privilege is claimed are defined as follows:

1. A compound having a carbohydrate moiety and an aglycone moiety and having the formula:

or a salt thereof,
wherein
L 2 is a linker comprising 1-20 carbon atoms in which one or more carbon atoms may be replaced with a heteroatom selected from N, O, and S, and/or one or more of carbon atoms may be substituted with a C 1 -C 6 alkyl group or halogen;
L 3 is a linker comprising 1-20 carbon atoms in which one or more carbon atoms may be replaced with a heteroatom selected from N, O, or S, and/or one or more of carbon atoms may be substituted with a C 1 -C 6 alkyl group or halogen;
L 4 is optional and when present is a linker comprising 1-20 carbon atoms in which one or more carbon atoms may be replaced with a heteroatom selected from N, O, or S), and/or one or more of carbon atoms may be substituted with a C 1 -C 6 alkyl group or halogen;
R 1 is a C 1 -C 10 alkyl group;
R 2 at each occurrence is independently selected from a C 1 -C 10 alkyl group, a C 1 -C 10 alkoxy group, halogen, nitro, —C(═O)NHR, or —C(═O)OR, where R is C 1 -C 8 alkyl group;
R 3 is a C 1 -C 10 alkyl group or a substituted or unsubstituted C 6 -C 10 aryl group; n is 0, 1,2, 3, or 4; and
S is

2. The compound of claim 1 , wherein L 2 is —(CH 2 ) n —, where n is 1-6.

3. The compound of claim 1 , wherein L 3 is —(CH 2 ) m —, where m is 1-12.

4. The compound of claim 1 , wherein L 4 is —(CH 2 ) n —, where n is 1-6.

5. The compound of claim 1 , wherein L 4 is absent.

6. The compound of claim 1 , wherein R 1 is C 1 -C 5 alkyl.

7. The compound of claim 1 , wherein R 2 is C 1 -C 8 alkyl.

8. The compound of claim 1 , wherein R 3 is C 1 -C 6 alkyl.

9. The compound of claim 1 , wherein R 3 is phenyl.

10. The compound of claim 1 having the formula:

or a salt thereof.

11. The compound of claim 1 having the formula:

or a salt thereof.

12. The compound of claim 1 having the formula:

or a salt thereof.

13. The compound of claim 1 having the formula:

or a salt thereof.

14. The compound of claim 1 having the formula:

or a salt thereof.

15. The compound of claim 1 having the formula:

or a salt thereof.

16. The compound of claim 1 having the formula:

or a salt thereof.

17. The compound of claim 1 having the formula:

or a salt thereof.

18. The compound of claim 1 having the formula:

or a salt thereof.

19. The compound of claim 1 having the formula:

or a salt thereof.

20. The compound of claim 1 having the formula:

or a salt thereof.

21. The compound of claim 1 having the formula:

or a salt thereof.

22. The compound of claim 1 having the formula:

or a salt thereof.

23. The compound of claim 1 having the formula:

or a salt thereof.

Assignees

• Univ Washington Through Its Center For Commercialization

Inventors

• Gelb Michael H
• Kumar Arun Babu
• Hocutt Frances
• Spacil Zdenek
• Barcenas Rodriguez Mariana Natali
• Turecek Frantisek
• Scott C Ronald

Classifications

• G01N2333/924

  acting on glycosyl compounds (3.2) · CPC title

• C12Q1/34 Primary

  involving hydrolase · CPC title

• G01N2333/916

  acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4) · CPC title

• C07H19/01

  sharing oxygen · CPC title

• C07H15/203 Primary

  Monocyclic carbocyclic rings other than cyclohexane rings; Bicyclic carbocyclic ring systems · CPC title