IP Library › Patent Application 16910727
Patent Application
App. No. 16/910,727

COMPOSITIONS AND METHODS

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Quick Facts
Patent No.
US None
App. No.
16/910,727
Abstract

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

Claims (18)

1 - 219 . (canceled)

220 . A compound comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide consists of 20 linked nucleosides and has the nucleobase sequence of any one of SEQ ID NOs: 78, 79, 80, and 82.

221 . The compound of claim 220 , wherein the conjugate group comprises:

222 . The compound of claim 220 , wherein the conjugate group is linked to the modified oligonucleotide at the 5′ end of the modified oligonucleotide.

223 . The compound of claim 220 , wherein the modified oligonucleotide comprises at least one modified sugar.

224 . The compound of claim 223 , wherein the at least one modified sugar is a bicyclic sugar.

225 . The compound of claim 224 , wherein the bicyclic sugar comprises a chemical modification selected from: 4′-(CH 2 )—O-2′ (LNA); 4′-(CH 2 ) 2 —O-2′ (ENA); and 4′-CH(CH 3 )—O-2′ (cEt).

226 . The compound of claim 223 , wherein the at least one modified sugar is 2′-O-methoxyethyl modified sugar.

227 . The compound of claim 220 , wherein the modified oligonucleotide comprises at least one modified nucleobase.

228 . The compound of claim 227 , wherein the modified nucleobase is a 5-methylcytosine.

229 . The compound of claim 220 , wherein the modified oligonucleotide comprises at least one modified internucleoside linkage.

230 . The compound of claim 229 , wherein the at least one modified internucleoside linkage is a phosphorothioate internucleoside linkage.

231 . The compound of claim 229 , wherein the modified oligonucleotide comprises at least one phosphodiester internucleoside linkage.

232 . A composition comprising the compound of claim 220 or a salt thereof, and at least one of a pharmaceutically acceptable carrier and diluent.

233 . A prodrug comprising the compound of claim 220 .

234 . A method comprising administering to an animal the compound of claim 220 .

235 . The method of claim 234 , wherein the animal is a human.

236 . The method of claim 234 , comprising co-administering the compound and a second agent.