The Scripps Research Institute;Cooley, Christina;Kelly, Jeffrey W.;Paxman, Ryan;Plate, Lars;Wiseman, R. Luke
发明人:
COOLEY, Christina,KELLY, Jeffery W.,PAXMAN, Ryan,PLATE, Lars,WISEMAN, R. Luke
申请号：
EP16882684
公开号：
EP3397256A4
申请日:
2016.12.29
申请国别(地区):
EP
年份:
2019
代理人:
摘要：
The invention provides compounds for activating the activating transcription factor 6 (ATF6) arm of the unfolded protein response (UPR), or activating the transcriptional targets of ATF6, in the endoplasmic reticulum of a cell, the compounds being of any of formulas (I) through (IX) as described herein. The compounds can be used for treatment of conditions involving gain-of-toxic-function and loss-of-function folding disorders including lysosomal storage diseases, antitrypsin-associated emphysema and similar diseases. These molecules are also expected to have disease-ameliorating effects in Alzheimer's disease and diabetes.
