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:
Nov. 19, 2019

Filed:

Oct. 09, 2015
Applicant:

Alnylam Pharmaceuticals, Inc., Cambridge, MA (US);

Inventors:

William Querbes, Boston, MA (US);

Kevin Fitzgerald, Brookline, MA (US);

Brian Bettencourt, Groton, MA (US);

Abigail Liebow, Somerville, MA (US);

David V. Erbe, Arlington, MA (US);

Assignee:

Alnylam Pharmaceuticals, Inc., Cambridge, MA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C07H 21/02 (2006.01); C07H 21/04 (2006.01); A61K 45/06 (2006.01); A61K 47/54 (2017.01); A61K 31/713 (2006.01); C12N 15/113 (2010.01); A61K 31/00 (2006.01); A61K 45/00 (2006.01);
U.S. Cl.
CPC ...
A61K 45/06 (2013.01); A61K 31/713 (2013.01); A61K 47/549 (2017.08); C12N 15/1137 (2013.01); A61K 31/00 (2013.01); A61K 45/00 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/344 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2310/3515 (2013.01); C12Y 101/03015 (2013.01);
Abstract

The invention relates to RNAi agents, e.g. double-stranded RNAi agents, targeting the HAO1 gene, and method of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1. Described herein are double-stranded RNAi agents which inhibit the expression of a HA01 gene in a cell, such as a cell within a subject, e.g., a mammal, such as a human having a HAO1 associated disorder, and uses of such double-stranded RNAi agents. In certain aspects of the invention, substantially all of the nucleotides of an iRNA of the invention are modified.


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