Compositions and methods
View Patent ↗Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
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.