Modulators of Complement Factor B
AssigneeIonis Pharmaceuticals, Inc.
InventorsTamar R. Grossman, Michael L McCaleb, Andrew T. Watt, Susan M. Freier
Life of this patent
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Abstract
The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
Claims25
A compound comprising a modified oligonucleotide consisting of 20 linked nucleosides having a nucleobase sequence consisting of the sequence recited in SEQ ID NO:
- 198, 228, 237, 444, 448, 450, or 453 wherein the modified oligonucleotide comprises:
- a gap segment consisting of ten linked deoxynucleosides;
- a 5′ wing segment consisting of five linked nucleosides;
- and
- a 3′ wing segment consisting of five 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 2′-O-methoxyethyl sugar;
- wherein each internucleoside linkage is a phosphorothioate linkage and wherein each cytosine is a 5-methylcytosine.
A compound comprising a modified oligonucleotide consisting of 16 linked nucleosides having a nucleobase sequence consisting of the sequence recited in SEQ ID NO:
- 550, 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
- the 5′ wing segment comprises a 2′-O-methoxyethyl sugar, 2′-O-methoxyethyl sugar, and cEt sugar in the 5′ to 3′ direction;
- wherein the 3′ wing segment comprises a cEt sugar, cEt sugar, and 2′-O-methoxyethyl sugar in the 5′ to 3′ direction;
- wherein each internucleoside linkage is a phosphorothioate linkage;
- and wherein each cytosine is a 5-methylcytosine.
A compound comprising a modified oligonucleotide consisting of 16 linked nucleosides having a nucleobase sequence consisting of the sequence recited in SEQ ID NO:
- 549, 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;
- and wherein each cytosine is a 5-methylcytosine.
A compound comprising a modified oligonucleotide consisting of 20 linked nucleosides having a nucleobase sequence consisting of the sequence recited in SEQ ID NO:
- 448, wherein the modified oligonucleotide comprises:
- a gap segment consisting of ten linked deoxynucleosides;
- a 5′ wing segment consisting of five linked nucleosides;
- and
- a 3′ wing segment consisting of five 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 2′-O-methoxyethyl sugar;
- wherein each internucleoside linkage is a phosphorothioate linkage and wherein each cytosine is a 5-methylcytosine.
Dependencies are inferred from the claim text.