Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease

a technology of glucoimidazole and phca, which is applied in the field of glucoimidazole (giz) and phca derivatives, can solve the problems of lack of specificity, difficult or inappropriate clinical use, complex protein folding, etc., and achieve the effect of enhancing the activity of gcas
US20050137223A1Inactive Publication Date: 2005-06-23AMICUS THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
AMICUS THERAPEUTICS INC
Publication Date
2005-06-23
Estimated Expiration
Not applicable · inactive patent

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Abstract

The present invention provides glucoimidazole (GIZ) and polyhydroxycyclohexenyl amine (PHCA) derivatives, methods of making them, and methods of use where the GIZ and PHCA derivatives have a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. More particularly, the present invention further provides a method for treating individuals having Gaucher disease by administering the novel GIZ or PHCA derivatives as “active-site specific chaperones” for the mutant glucocerebrosidase associated with the disease.
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Description

[0001] This application claims priority to provisional application 60 / 519,496 filed Nov. 12, 2003, herein incorporated by reference.FIELD OF THE INVENTION

[0002] The present invention provides novel glucoimidazole (GIZ) and polyhydroxy-cyclohexenyl amine (PHCA) derivatives having a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel GIZ or PHCA derivatives as “active-site specific chaperones” for the mutant glucocerebrosidase associated with the disease. BACKGROUND OF THE INVENTION Protein Folding

[0003] Proteins are synthesized in the cytoplasm, and the newly synthesized proteins are secreted into the lumen of the endoplasmic reticulum (ER) in a largely unfolded state. In general, protein folding is governed by the princ...

Claims

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