IP Library Granted Patent US 11,174,481
Granted Patent B2
US 11,174,481 · App. 16/380,695 · Granted Nov 16, 2021

Targeting ligands

Inventors: Zhen Li (Monona, WI); Tao Pei (Monona, WI); Agnieszka Glebocka (Madison, WI); Michael Lawler (Madison, WI); Fred Fleitz (Germantown, WI); Erich Altenhofer (Madison, WI); Pankaj Kumar (Madison, WI)
Assignee: Arrowhead Pharmaceuticals, Inc.
C12N15/113A61K31/7008A61K31/711A61K31/713A61K31/7105A61K47/549A61K47/555C07H5/06C07H21/02C07H21/04C12N2310/14C12N2310/31C12N2310/315C12N2310/317C12N2310/321C12N2310/322C12N2310/332C12N2310/351C12N2310/3515C12N2310/3517C12N2320/32
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Quick Facts
Patent No.
US 11,174,481
App. No.
16/380,695
Granted
Nov 16, 2021
Kind
B2
Abstract

Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target. 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 a therapeutic 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.

Claims (5)

1. A compound having a structure selected from the group consisting of:

or a pharmaceutically acceptable salt thereof, wherein R is an RNAi agent, Y is O or S, and Y′ is 0 ″, S″, or NH″.

2. The compound of claim 1 , wherein Y is S.

3. The compound of claim 1 , wherein Y is O.

4. The compound of claim 1 , wherein Y′ is 0 ″.

Assignments (2)
SECURITY INTEREST Recorded Aug 7, 2024
From: ARROWHEAD PHARMACEUTICALS, INC.
To: SIXTH STREETLENDING PARTNERS, AS THE ADMINISTRATIVE AGENT
Reel/Frame 068510/0363 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 17, 2020
From: LI, ZHEN; PEI, TAO; GLEBOCKA, AGNIESZKA; LAWLER, MICHAEL; FLEITZ, FRED; ALTENHOFER, ERICH; KUMAR, PANKAJ
To: ARROWHEAD PHARMACEUTICALS, INC.
Reel/Frame 053509/0457 →
Continuity (4)
Continuation 15452324 · Mar 7, 2017
Provisional Application 62456339 · Feb 8, 2017
Provisional Application 62383221 · Sep 2, 2016
Related Publication 20190256849A1 · Aug 22, 2019