Patent ID: 9610358
Date: 2017-04-04
CPC Classifications: A61K,C07K,C12Q,G01N,Y02A

Claim:
1. A method of treating a lysosomal storage disease in a subject, comprising: A. preparing a targeted pharmacological chaperone having the general formula: where T represents a targeting group that is a peptide having an amino acid sequence selected from the group consisting of SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:9, SEQ ID NO:10, SEQ ID NO:11, SEQ ID NO:12, SEQ ID NO:13, SEQ ID NO:14, SEQ ID NO:15, SEQ ID NO:16, SEQ ID NO:17, SEQ ID NO:18, SEQ ID NO:19, SEQ ID NO:20, SEQ ID NO:21, SEQ ID NO:22, SEQ ID NO:23, SEQ ID NO:24, and SEQ ID NO:25; whew DRUG represents a small molecule compound selected from the group consisting of nojirimycins, alkyl Glucosamines, and thioglycosides; where LINK represents an optional linking group having 1-20 non-hydrogen atoms selected from the group consisting of C, N, O and S composed of any combination of chemical bonds, including ether, thioether, succinylthioether, benzylthioether, amine, ester, carboxamide, sulfonamide, hydrazide bonds, and single, double, triple carbon-carbon bonds, and aromatic or heteroaromatic bonds; and B. administering a therapeutically effective amount of said targeted pharmacological chaperone to a subject suffering from a lysosomal storage disease.