Modulators of PCSK9 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsEric E. Swayze, Susan M. Freier
Life of this patent
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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.
Claims10
A compound according to the following formula (SEQ ID NO:
- 1016):
- or a pharmaceutically acceptable salt thereof.
A compound or a pharmaceutically acceptable salt thereof, comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide is 16 linked nucleosides in length and has the nucleobase sequence consisting of the nucleobase sequence of SEQ ID NO:
- 1016, wherein the modified oligonucleotide has
- a gap segment consisting of ten linked deoxynucleosides;
- a 5′ wing segment consisting of three linked nucleosides;
- and
- a 3′ wing segment consisting of three linked nucleosides;
- wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, wherein each nucleoside of each wing segment comprises a cEt sugar;
- wherein each internucleoside linkage is a phosphorothioate linkage;
- wherein each cytosine is a 5-methylcytosine.
A compound consisting of a modified oligonucleotide, wherein the modified oligonucleotide is 16 linked nucleosides in length and consists of the sequence of SEQ ID NO:
- 1016, wherein the modified oligonucleotide consists
- of a gap segment consisting of ten linked deoxynucleosides;
- a 5′ wing segment consisting of three linked nucleosides;
- and
- a 3′ wing segment consisting of three linked nucleosides;
- wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, wherein each nucleoside of each wing segment comprises a cEt sugar;
- wherein each internucleoside linkage is a phosphorothioate linkage;
- and wherein each cytosine is a 5-methylcytosine.
Dependencies are inferred from the claim text.