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US 2022/0023429 A1Published application
Conjugated antisense compounds and their use
AssigneeIonis Pharmaceuticals, Inc.
InventorsPunit P. Seth, Michael Oestergaard, Eric E. Swayze
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Abstract
The present disclosure provides duplexes comprising a first oligomeric compound and a second oligomeric compound wherein the second oligomeric compound comprises a conjugate group. In certain embodiments, the duplex modulates the amount or activity of a target nucleic acid in extra hepatic tissues and/or extra hepatic cells. In certain embodiments, the duplex modulates the amount or activity of a target nucleic acid in hepatic tissues and/or hepatic cells.
Claims25
1-377. (canceled)
A duplex comprising a first oligomeric compound and a second oligomeric compound wherein:
- the first oligomeric compound comprises a first modified oligonucleotide consisting of 10-30 linked nucleosides, having a nucleobase sequence that is at least 80% complementary to the nucleobase sequence of the second oligomeric compound and that is at least 80% complementary to an extra-hepatic nucleic acid target;
- wherein neither the first oligomeric compound nor the duplex is an RNAi compound;
- the second oligomeric compound comprises a conjugate group and a second modified oligonucleotide consisting of 10-30 linked nucleosides;
- wherein the conjugate group comprises a conjugate moiety and a conjugate linker,
- wherein the conjugate moiety is cholesterol;
- and wherein the conjugate linker comprises at least one cleavable moiety;
- and
- wherein the extra-hepatic nucleic acid target is expressed in the brain or spinal cord.
5 more elements
Dependencies are inferred from the claim text.