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US 11,633,483 B2Granted
Modulators of PCSK9 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsEric E. Swayze, Susan M. Freier, Huynh-Hoa Bui
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Abstract
The present embodiments provide methods, compounds, and compositions useful for inhibiting PCSK9 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PCSK9.
Claims15
An oligomeric compound comprising a modified oligonucleotide according to the following formula:
- Aks Aks Tks Ads Ads Tds mCds Tds mCds Ads Tds Gds Tds mCks Aks Gk (SEQ ID NO:
- 1016);
- wherein,
- A=an adenine,
- mC=a 5-methylcytosine,
- G=a guanine,
- T=a thymine,
- k=a cEt modified nucleoside,
- d=β-D-2′-deoxyribonucleoside, and
- s=a phosphorothioate internucleoside linkage.
7 more elements
An oligomeric compound comprising a modified oligonucleotide and a conjugate group according to the following formula:
- GalNAc3-7a-o, Aks Aks Tks Ads Ads Tds mCds Tds mCds Ads Tds Gds Tds mCks Aks Gk (SEQ ID NO:
- 1016);
- wherein,
- A=an adenine,
- mC=a 5-methylcytosine,
- G=a guanine,
- T=a thymine,
- k=a cEt modified nucleoside,
- d=β-D-2′-deoxyribonucleoside,
- s=a phosphorothioate internucleoside linkage, and
- wherein the cleavable moiety (CM) comprises a phosphodiester bond.
8 more elements
Dependencies are inferred from the claim text.