US2021284680A1PendingUtilityA1

Antisense nucleic acids

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Assignee: NIPPON SHINYAKU CO LTDPriority: Sep 1, 2010Filed: May 28, 2021Published: Sep 16, 2021
Est. expirySep 1, 2030(~4.1 yrs left)· nominal 20-yr term from priority
Inventors:Naoki WatanabeYouhei SatouShin'Ichi TakedaTetsuya Nagata
A61K 31/7125A61P 21/00C07H 21/04C12N 15/113C12N 2310/11C07H 21/00C12N 2320/33C12N 2310/321C07K 14/4708C12N 2310/3525C12N 15/111C12N 2310/315C12N 2310/3145
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Cited by
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Claims

Abstract

The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.

Claims

exact text as granted — not AI-modified
1 - 14 . (canceled) 
     
     
         15 . A composition comprising an antisense oligomer having the formula: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
       and a pharmaceutically acceptable additive. 
     
     
         16 . The composition of  claim 15 , wherein the pharmaceutically acceptable additive is sodium chloride. 
     
     
         17 . The composition of  claim 15 , wherein the antisense oligomer comprises a carrier. 
     
     
         18 . The composition of  claim 17 , wherein the carrier is a cationic polymer. 
     
     
         19 . The composition of  claim 17 , wherein the carrier promotes delivery of the antisense oligomer to a muscle tissue.

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