Back to results
US 2026/0250680 A1Published application
Conjugated antisense compounds and their use
AssigneeIonis Pharmaceuticals, Inc.
InventorsThazha P. Prakash, Punit P. Seth, Eric E. Swayze
Life of this patent
Estimating the term…
Abstract
Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
Claims20
1-296. (canceled)
A conjugated oligonucleotide comprising an oligonucleotide and a conjugate group, wherein the conjugate group comprises:
- (i) at least one phosphorous linking group or neutral linking group;
- (ii) three ligands wherein the ligand is N-Acetyl galactosamine;
- (iii) a branching group having the structure
- and
- (iv) three tethers, wherein one ligand is covalently attached to each tether, and each tether is covalently attached to the branching group,
- wherein the oligonucleotide comprises at least one modified nucleoside;
- and
- wherein the oligonucleotide comprises an antisense strand having a nucleobase sequence comprising an at least 8 contiguous nucleobase portion complementary to an equal length contiguous portion of a target nucleic acid, wherein the target nucleic encodes a protein selected from among:
- Androgen Receptor, Apolipoprotein (a), Apolipoprotein B, Apolipoprotein C-III, C-Reactive Protein, eIF-4E, Factor VII, Factor XI, Glucocorticoid Receptor, Glucagon Receptor, Protein Tyrosine Phosphatase 1B, STAT3, SRB-1, and Transthyretin.
6 more elements
Dependencies are inferred from the claim text.