The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Jan. 30, 2024

Filed:

Nov. 10, 2021
Applicant:

Arrowhead Pharmaceuticals, Inc., Pasadena, CA (US);

Inventors:

Zhen Li, Westfield, NJ (US);

Rui Zhu, Middleton, WI (US);

Christine I. Wooddell, Madison, WI (US);

Tao Pei, Middleton, WI (US);

Assignee:

Arrowhead Pharmaceuticals, Inc., Pasadena, CA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 9/00 (2006.01); A61P 1/16 (2006.01); G01N 33/68 (2006.01); C12Q 1/6883 (2018.01); A61K 31/713 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 9/0019 (2013.01); A61K 31/713 (2013.01); A61P 1/16 (2018.01); C12Q 1/6883 (2013.01); G01N 33/6893 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/317 (2013.01); C12N 2310/32 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/333 (2013.01); C12N 2310/334 (2013.01); C12N 2310/335 (2013.01); C12N 2310/336 (2013.01); C12N 2310/343 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2320/31 (2013.01); C12Q 2600/156 (2013.01); C12Q 2600/158 (2013.01); G01N 2333/8125 (2013.01); G01N 2800/085 (2013.01);
Abstract

RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.


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