Universitéde Montreal

Amicus Therapeutics, Inc.

Patricia René

Michel Bouvier

Anne Marinier

Jian-Qiang Fan

Gary Lee

Kenneth Valenzano

Stéphane Gingras

Réjean Ruel

Philippe Dagneau

Rejean Ruel

Yves Andre Chantigny

Yves Chantigny

Ken Vanlenzano

The invention relates to methods of enhancing normal melanocortin-4 receptor (MC4R) activity, and to enhancing activity of an MC4R having a mutation which affects protein folding and/or processing of the MC4R. The invention provides a method of treating an individual having a condition in which increased activity of an MC4R at the cell surface would be beneficial, for example in obesity, by administering an effective amount of a pharmacological chaperone for the MC4R. The invention provides MC4R pharmacological chaperones which enhance the activity of MC4R. The invention further provides a method of screening to identify pharmacological chaperones which enhance folding of an MC4R in the endoplasmic reticulum (ER), in order to enhance the activity of the MC4R at the cell surface.

Pharmacological chaperones for treating obesity

Disclosed herein is a compound of Formula I: 

Pyrazolopyridine and pyrazolopyrimidine derivatives as melanocortin-4 receptor modulators

Disclosed herein is a compound of Formula I: wherein X, R, R, and Rare as defend herein, or a pharmaceutically acceptable salt thereof to allow the drug to penetrate the cell membrane; or a prodrug, or the compound is labeled with a detectable label or an affinity tag thereof. Also disclosed is a pharmaceutical composition, a method of treating a disorder mediated by melanocortin-4 receptors, and a method of treating obesity using the compounds described. 

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Montréal, Canada

Patricia René