公开(公告)号：WO2022060721A2

公开(公告)日：2022-03-24

申请号：PCT/US2021/050247

申请日：2021-09-14

申请人： ARROWHEAD PHARMACEUTICALS, INC.

发明人： CHRISTIANSON, Dawn ,  HAMILTON, James C. ,  GIVEN, Bruce ,  SAN MARTIN, Javier

IPC分类号： C12N15/113 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2320/35

摘要： Described are methods of reducing liver Z-AAT protein levels in a human subject with a PiZZ genotype of alpha-1 antitrypsin (AAT) by using pharmaceutical compositions that include AAT RNAi agents. The pharmaceutical compositions disclosed herein that include AAT RNAi agents, when administered to a human subject with a PiZZ mutation, lead to a reduction in liver Z-AAT protein levels, including both soluble and insoluble Z-AAT protein. Such reductions can lead to the treatment of liver diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, fulminant hepatic failure, and other liver-related diseases.

公开(公告)号：WO2020247774A1

公开(公告)日：2020-12-10

申请号：PCT/US2020/036359

申请日：2020-06-05

申请人： ARROWHEAD PHARMACEUTICALS, INC.

发明人： GIVEN, Bruce ,  CHRISTIANSON, Dawn ,  HAMILTON, James ,  LI, Zhen ,  ZHU, Rui ,  WOODDELL, Christine ,  PEI, Tao

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K31/712 ,  A61K31/7125 ,  A61P1/16 ,  A61P11/00

摘要： Described are methods for treating alpha- 1 antitrypsin deficiency (AATD) in a human patient in need of treatment, using pharmaceutical compositions that include AAT RNAi agents. The pharmaceutical compositions disclosed herein that include AAT RNAi agents, when administered to a human patient in need thereof, treat liver diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, fulminant hepatic failure, and other liver-related diseases.