公开(公告)号：EP2933265B1

公开(公告)日：2018-11-14

申请号：EP15153180.3

申请日：2006-06-05

申请人： Amicus Therapeutics, Inc. ,  Université de Montréal

发明人： FAN, Jian-qiang ,  VANLENZANO, Ken ,  LEE, Gary ,  BOUVIER, Michel ,  RENÉ, Patricia

IPC分类号： C07K14/72 ,  C12N5/071 ,  A61K31/497 ,  A61K31/505 ,  A61K31/4164

CPC分类号： A61K31/495 ,  A61K9/0053 ,  A61K31/497 ,  A61K31/505 ,  C07K14/723

摘要： 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.

公开(公告)号：EP1885366A2

公开(公告)日：2008-02-13

申请号：EP06772163.9

申请日：2006-06-05

申请人： Amicus Therapeutics, Inc.

发明人： FAN, Jian-qiang ,  VANLENZANO, Ken ,  LEE, Gary ,  BOUVIER, Michel Universite de Montreal

IPC分类号： A61K31/497 ,  A61K31/505

CPC分类号： A61K31/495 ,  A61K9/0053 ,  A61K31/497 ,  A61K31/505 ,  C07K14/723

摘要： 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.