Method for treating cerebral amyloid angiopathy using pharmacological chaperones to increase the activity of gangliosidases

• US 9,931,353 B2
• Filed: 05/05/2015
• Issued: 04/03/2018
• Est. Priority Date: 10/19/2009
• Status: Active Grant

• Current Assignee: Amicus Therapeutics, Inc.
• Original Assignee: Amicus Therapeutics, Inc.
• Inventors: Wustman, Brandon Alan, Valenzano, Kenneth, Boyd, Robert
• Primary Examiner(s): MCMILLIAN, KARA RENITA
• Application Number:
  US14/704,445
• Publication Number:
  US 20150374733A1
• Time in Patent Office: 1,064 Days
• CPC Class Codes: A61K 31/426 1,3-Thiazoles
• CPC Class Codes: A61K 31/429 condensed with heterocyclic ring systems
• CPC Class Codes: A61K 31/445 Non condensed piperidines, e.g. piperocaine
• CPC Class Codes: A61K 31/7008 Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
• CPC Class Codes: A61K 38/1709 from mammals
• CPC Class Codes: A61P 25/28 for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Abstract

The present invention relates to a method for treating an individual having Cerebral Amyloid Angiopathy by using pharmacological chaperones to increase the activity of gangliosidase and/or sialidase enzymes involved in ganglioside catabolism.

Claims

1. A method for the treatment of Cerebral Amyloid Angiopathy in an individual, comprising administering to the individual an effective amount of a pharmacological chaperone, wherein the pharmacological chaperone is selected from 2-acetamido-1,2-dideoxynojirimycin and N-acetyl-glucosamine-thiazoline.

2. The method of claim 1, wherein the pharmacological chaperone increases production of sAPPα

.

3. The method of claim 1, wherein the pharmacological chaperone is 2-acetamido-1,2-dideoxynojirimycin.

4. The method of claim 1, wherein the pharmacological chaperone is N-acetylglucosamine-thiazoline.

5. The method of claim 1, wherein the Cerebral Amyloid Angiopathy is Familial Cerebral Amyloid Angiopathy.

6. The method of claim 5, wherein the Familial Cerebral Amyloid Angiopathy is caused by a mutation in amyloid precursor protein (APP).