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1. (WO2016057893) COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION

Pub. No.:    WO/2016/057893    International Application No.:    PCT/US2015/054881
Publication Date: Fri Apr 15 01:59:59 CEST 2016 International Filing Date: Sat Oct 10 01:59:59 CEST 2015
IPC: A61K 31/713
Applicants: ALNYLAM PHARMACEUTICALS, INC.
Inventors: QUERBES, William
FITZGERALD, Kevin
BETTENCOURT, Brian
LIEBOW, Abigail
ERBE, David, V.
Title: COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
Abstract:
The invention relates to RNAi agents, e.g. double-stranded RNAi agents, targeting the HAO1 gene, and methods 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.