Modulators of PNPLA3 expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsSusan M. Freier, Huynh-Hoa Bui
Life of this patent
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Abstract
The present embodiments provide methods, compounds, and compositions useful for inhibiting PNPLA3 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PNPLA3.
Claims9
A compound, wherein an anionic form of the compound has the following formula (SEQ ID NO:
- 1089):
- or a pharmaceutically acceptable salt thereof.
A modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide is 16 linked nucleosides in length and has the nucleobase sequence of SEQ ID NO:
- 1089, wherein the modified oligonucleotide comprises:
- 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;
- and wherein the conjugate group is positioned at the 5′ end of the modified oligonucleotide and is
A compound, wherein an anionic form of the compound has the following formula (SEQ ID NO:
- 1089):
- or a pharmaceutically acceptable salt thereof.
A modified oligonucleotide, and wherein the modified oligonucleotide is conjugated to a conjugate group, wherein the modified oligonucleotide is 16 linked nucleosides in length and has the nucleobase sequence of SEQ ID NO:
- 1089, wherein the modified oligonucleotide comprises:
- 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 at least one internucleoside linkage is a phosphorothioate linkage;
- wherein each cytosine is a 5-methylcytosine.
A compound comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide is 16 linked nucleosides in length and has the nucleobase sequence of SEQ ID NO:
- 1089, wherein the modified oligonucleotide comprises:
- 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 modified oligonucleotide, wherein the modified oligonucleotide consists of 16 linked nucleosides and has the nucleobase sequence consisting of SEQ ID NO:
- 1089, wherein the modified oligonucleotide comprises:
- 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.
Dependencies are inferred from the claim text.