IP Library Granted Patent US 12,116,573
Granted Patent B2
US 12,116,573 · App. 17/184,575 · Granted Oct 15, 2024

Targeting ligands for therapeutic compounds

Inventors: David B. Rozema (Middleton, WI); Darren H. Wakefield (Fitchburg, WI); Andrei V. Blokhin (Fitchburg, WI); Jonathan D. Benson (Madison, WI); Zhen Li (Westfield, NJ); Tao Pei (Monona, WI); Fred Fleitz (Pasadena, CA)
Assignee: Arrowhead Pharmaceuticals, Inc.
C12N15/113A61K31/713A61K47/549C07H15/04C12N2310/14C12N2310/317C12N2310/32C12N2310/321C12N2310/322C12N2310/351C12N2310/3515
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Quick Facts
Patent No.
US 12,116,573
App. No.
17/184,575
Granted
Oct 15, 2024
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 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.

Claims (4)

1. A targeting ligand selected from the group consisting of:

wherein Targeting Moiety is selected from the group consisting of: carbohydrates, glycans, haptens, vitamins, folate, biotin, aptamers, peptides, insulin, EGF and transferrin.

2. The targeting ligand of claim 1 , wherein the Targeting Moiety is a carbohydrate.

3. The targeting ligand of claim 1 , wherein the targeting ligand is selected from the group consisting of:

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 Feb 25, 2021
From: ROZEMA, DAVID B; WAKEFIELD, DARREN H; BLOKHIN, ANDREI V; BENSON, JONATHAN D; LI, ZHEN; PEI, TAO; FLEITZ, FRED
To: ARROWHEAD PHARMACEUTICALS, INC.
Reel/Frame 055416/0927 →