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1. (WO2017156012) TARGETING LIGANDS FOR THERAPEUTIC COMPOUNDS

Pub. No.:    WO/2017/156012    International Application No.:    PCT/US2017/021175
Publication Date: Fri Sep 15 01:59:59 CEST 2017 International Filing Date: Wed Mar 08 00:59:59 CET 2017
IPC: A61K 31/713
C12N 15/11
C12N 15/87
Applicants: ARROWHEAD PHARMACEUTICALS, INC.
Inventors: ROZEMA, David, B.
WAKEFIELD, Darren, H.
BLOKHIN, Andrei, V.
BENSON, Jonathan, D.
LI, Zhen
PEI, Tao
FLEITZ, Fred
Title: TARGETING LIGANDS FOR THERAPEUTIC COMPOUNDS
Abstract:
Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds, that are useful in directing the compounds to the target in vivo. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to an expression-inhibiting oligomeric compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.