US2022288100A1PendingUtilityA1

2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations

Assignee: IONIS PHARMACEUTICALS INCPriority: Nov 5, 2002Filed: Nov 3, 2021Published: Sep 15, 2022
Est. expiryNov 5, 2022(expired)· nominal 20-yr term from priority
C12N 2310/342C12N 15/111A61K 31/713C12N 2310/315C12N 2310/344C12N 2310/14
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Claims

Abstract

Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2′-OCH3 substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.

Claims

exact text as granted — not AI-modified
1 . A method for assessing the activity of an oligomeric compound comprising contacting the oligomeric compound with a cell in vitro, wherein the oligomeric compound comprises a first oligomer and a second oligomer, wherein:
 at least a portion of said first oligomer is capable of hybridizing with at least a portion of said second oligomer,   at least a portion of said first oligomer is complementary to and capable of hybridizing with a selected target nucleic acid,   each of said first and second oligomers is a chimeric oligomer,   
       wherein the second oligomer has an internal segment comprising at least four consecutive nucleosides each having a 2′-OCH 3  substituent group, and 
       wherein the chimeric oligomers are not a gapmers; 
       measuring or detecting a phenotypic change in the cell in vitro; and 
       thereby assessing the activity of the oligomeric compound. 
     
     
         2 .- 19 . (canceled)

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