Massachusetts Institute of Technology;Yale University
发明人:
Bogdan I. Fedeles,Sorin V. Fedeles,Robert G. Croy,Stefan Somlo,John M. Essigmann
申请号：
US15953168
公开号：
US20180362574A1
申请日:
2018.04.13
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention provides compounds of Formula (I) or (II), which are thought to be able to inhibit mTOR (mammalian target of rapamycin) signaling pathway, induce UPR (unfolded protein response), and/or perturb mitochondrial function of a cyst cell (e.g., a cyst cell causing polycystic kidney disease (PKD, e.g., autosomal dominant PKD (ADPKD) or autosomal recessive PKD (ARPKD)) or polycystic liver disease (PLD, e.g., autosomal dominant PLD (ADPLD) or autosomal recessive PLD (ARPLD)). The invention also provides pharmaceutical compositions, kits, and methods involving the compounds described herein for use in treating PKD or PLD, inhibiting the growth of a cyst cell, and/or killing a cyst cell.
