Conjugated antisense compounds and their use
AssigneeIonis Pharmaceuticals, Inc.
InventorsMichael Oestergaard, Punit P. Seth, Frank Rigo, Chrissa A. Dwyer
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Abstract
The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.
Claims18
A compound comprising a first oligomeric compound and a second oligomeric compound, wherein the first oligomeric compound comprises a first modified oligonucleotide consisting of 14-30 linked nucleosides and has a nucleobase sequence complementary to the nucleobase sequence of the second oligomeric compound and to a nucleic acid target;
- and
- the second oligomeric compound comprises a second modified oligonucleotide consisting of 6-12 linked nucleosides;
- wherein the first modified oligonucleotide has a sugar motif comprising:
- a 5′-region consisting of 1-5 linked 5′-nucleosides,
- a central region consisting of 6-10 linked central region nucleosides, and
- a 3′-region consisting of 1-5 linked 3′-nucleosides;
- wherein each of the 5′-region nucleosides and each of the 3′-region comprises a modified sugar moiety and each of the central region nucleosides comprises an unmodified DNA sugar moiety.
Dependencies are inferred from the claim text.