US2021130823A1PendingUtilityA1

Compositions and methods

78
Assignee: IONIS PHARMACEUTICALS INCPriority: May 1, 2013Filed: Jun 24, 2020Published: May 6, 2021
Est. expiryMay 1, 2033(~6.8 yrs left)· nominal 20-yr term from priority
Inventors:Thazha P. PrakashPunit P. SethEric E. Swayze
C12N 15/113C12N 2310/315C12N 2310/11A61K 31/713A61K 31/7088C07H 21/00C12N 2310/35C12N 2310/32A61K 48/00A61K 31/7125C12N 2310/34C12N 2310/3525C12N 2310/14A61K 31/7105C12N 15/1137A61P 25/00A61P 43/00A61K 47/59C12Y 301/03048C12N 2310/322C12N 2310/321A61K 47/549C12N 2310/353C12N 2310/113C12N 2310/31C07H 21/04C12N 2310/351C12N 2310/3341C12N 15/111C12N 2310/341C12N 2310/17C12N 2310/3231C12N 2320/32C12N 2310/346C12N 2310/3515C12N 2310/3513C12N 2310/3511
78
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Claims

Abstract

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

Claims

exact text as granted — not AI-modified
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.

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