US2019276823A1PendingUtilityA1

Selective antisense compounds and uses thereof

Assignee: IONIS PHARMACEUTICALS INCPriority: Oct 12, 2012Filed: Sep 25, 2018Published: Sep 12, 2019
Est. expiryOct 12, 2032(~6.2 yrs left)· nominal 20-yr term from priority
C12N 2310/345C07H 21/02C12N 2310/346A61P 25/14A61P 25/28C12N 2310/3125C12N 2310/335C12N 2310/3231C12N 2310/3341C12N 2320/34C12N 2310/11C12N 2310/313C12N 2310/341C12N 2310/315C12N 15/113
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Claims

Abstract

The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

Claims

exact text as granted — not AI-modified
1 .- 40 . (canceled) 
     
     
         41 . A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the linked nucleosides comprise at least 8 contiguous nucleobases of a nucleobase sequence recited in SEQ ID NO: 5, 6, 27, 28, 29, 30, 38, 39, or 46. 
     
     
         42 . The compound of  claim 41 , wherein the nucleobase sequence of the modified oligonucleotide is 100% complementary to SEQ ID NO. 1. 
     
     
         43 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 5. 
     
     
         44 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 6. 
     
     
         45 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 27. 
     
     
         46 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 28. 
     
     
         47 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 29. 
     
     
         48 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 30. 
     
     
         49 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 38. 
     
     
         50 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 39. 
     
     
         51 . The compound of  claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 46. 
     
     
         52 . The compound of  claim 43 , wherein the oligonucleotide has a ekek-d9-keke sugar motif. 
     
     
         53 . The compound of  claim 44 , wherein the oligonucleotide has a kekk-d8-keke sugar motif. 
     
     
         54 . The compound of  claim 45 , wherein the oligonucleotide has a ekkk-d8-kke sugar motif. 
     
     
         55 . The compound of  claim 46 , wherein the oligonucleotide has a ekk-d8-kkke sugar motif. 
     
     
         56 . The compound of  claim 47 , wherein the oligonucleotide has a ekekk-d8-keke sugar motif. 
     
     
         57 . The compound of  claim 48 , wherein the oligonucleotide has a ekek-d8-kkeke sugar motif. 
     
     
         58 . The compound of  claim 49 , wherein the oligonucleotide has a eekk-d8-kkeee sugar motif. 
     
     
         59 . The compound of  claim 50 , wherein the oligonucleotide has a eekk-d8-kkeee sugar motif. 
     
     
         60 . The compound of  claim 51 , wherein the oligonucleotide has a eeekk-d8-kkee sugar motif. 
     
     
         61 . The compound of  claim 41 , comprising at least one phosphorothioate internucleoside linkage. 
     
     
         62 . A method of ameliorating a symptom of Huntington's disease, comprising administering an oligomeric compound of  claim 41  to an animal in need thereof.

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