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:
Aug. 06, 2024

Filed:

Mar. 26, 2021
Applicant:

Arrowhead Pharmaceuticals, Inc., Madison, WI (US);

Inventors:

Anthony Nicholas, Oregon, WI (US);

Tao Pei, Middleton, WI (US);

Zhao Xu, Brookfield, WI (US);

Casi Schienebeck, Deerfield, WI (US);

Zhi-Ming Ding, Waunakee, WI (US);

Assignee:

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

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 47/54 (2017.01); A61P 1/16 (2006.01);
U.S. Cl.
CPC ...
C12N 15/1137 (2013.01); A61K 47/549 (2017.08); A61P 1/16 (2018.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2320/35 (2013.01);
Abstract

Described are RNAi agents, e.g., double stranded RNAi agents, for inhibiting patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Also disclosed are pharmaceutical compositions that include PNPLA3 RNAi agents and methods of use thereof. The PNPLA3 RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Delivery of the PNPLA3 RNAi agents in vivo provides for inhibition of PNPLA3 gene expression. The RNAi agents can be used in methods of treatment of PNPLA3-related diseases and disorders, including non-alcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), hepatic fibrosis, and alcoholic or non-alcoholic liver diseases, including cirrhosis.


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