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US 2026/0022382 A1Published application
Compositions and Methods for Modulating Complement Factor B Expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsThazha P. Prakash, Punit P. Seth, Eric E. Swayze, Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
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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.
Claims2
1-263. (canceled)
A compound comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide consists of 10 to 30 linked nucleosides having a nucleobase sequence comprising any one of SEQ ID NOs:
- 198, 228, 237, 440, 444, 448, 450, 453, 455, 549, and 598, wherein the modified oligonucleotide comprises:
- a gap segment consisting of linked deoxynucleosides;
- a 5′ wing segment consisting of linked nucleosides;
- and
- a 3′ wing segment consisting of linked nucleosides;
- wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment and
- wherein each nucleoside of each wing segment comprises a modified sugar.
4 more elements
Dependencies are inferred from the claim text.