Methods for the treatment of alpha-1 antitrypsin deficiency (AATD)
Grant
US12582668B2
Kind: B2
Mar 24, 2026
Assignee
Arrowhead Pharmaceuticals, Inc.
Inventors
Bruce Given, Dawn Christianson, James C. Hamilton, Zhen Li, Rui Zhu, Christine I. Wooddell, Tao Pei
Abstract
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.
CPC Classifications
A61K 31/713
A61K 9/0019
A61K 48/00
C12N 15/113
C12N 2310/346
C12N 2310/315
Filing Date
2020-06-05
Application No.
17616292
Claims
22