IP Library Granted Patent US 9,714,421
Granted Patent B2
US 9,714,421 · App. 14/888,318 · Granted Jul 25, 2017

Compositions and methods

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Quick Facts
Patent No.
US 9,714,421
App. No.
14/888,318
Granted
Jul 25, 2017
Kind
B2
Abstract

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

Claims (18)

1. A compound comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide is 10-30 linked nucleosides in length and consists of the nucleobase sequence of SEQ ID NO: 81, wherein the conjugate group comprises:

and wherein the conjugate group is linked to the modified oligonucleotide at the 5′ end of the modified oligonucleotide.

2. The compound of claim 1 , wherein the modified oligonucleotide comprises at least one modified sugar.

3. The compound of claim 2 , wherein the modified sugar is a bicyclic sugar.

4. The compound of claim 3 , wherein the bicyclic sugar is selected from the group consisting of: 4′-(CH 2 )—O-2′ (LNA); 4′-(CH 2 ) 2 —O-2′ (ENA); and 4′-CH(CH 3 )—O-2′ (cEt).

5. The compound of claim 2 , wherein the modified sugar is 2′-O-methoxyethyl.

6. The compound of claim 1 , wherein the modified oligonucleotide comprises at least one modified nucleobase.

7. The compound of claim 6 , wherein the modified nucleobase is a 5-methylcytosine.

8. The compound of claim 1 , wherein the compound is single-stranded.

9. The compound of claim 1 , wherein the compound is double-stranded.

10. The compound of claim 1 , wherein the modified oligonucleotide comprises at least one modified internucleoside linkage.

11. The compound of claim 10 , wherein the modified internucleoside linkage is a phosphorothioate internucleoside linkage.

12. The compound of claim 10 , wherein the modified oligonucleotide comprises at least one phosphodiester internucleoside linkage.

13. A composition comprising the compound of claim 1 or salt thereof and at least one of a pharmaceutically acceptable carrier or diluent.

14. A prodrug comprising the compound of claim 1 .

15. A method comprising administering to an animal the compound or composition of claim 1 .

16. The method of claim 15 , wherein the animal is a human.

17. The method of claim 15 , comprising co-administering the compound or composition and a second agent.

Assignments (1)
CHANGE OF NAME Recorded Feb 18, 2016
From: ISIS PHARMACEUTICALS, INC.
To: IONIS PHARMACEUTICALS, INC.
Reel/Frame 037847/0719 →