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US 2024/0076676 A1Published application
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.
Claims29
1-7. (canceled)
A compound comprising a modified oligonucleotide 10 to 30 linked nucleosides in length, wherein the modified oligonucleotide has a nucleobase sequence comprising any one of SEQ ID NOs:
- 1089, 1757, 141, 1982, 330, 1665, 408, 830, and 899, wherein the modified oligonucleotide is at least 80% complementary to SEQ ID NO:
- 2 over the entire length of the modified oligonucleotide, and wherein the modified oligonucleotide comprises at least one modification selected from at least one modified internucleoside linkage, at least one modified sugar, and at least one modified nucleobase.
9-11. (canceled)
18-22. (canceled)
25-26. (canceled)
29-31. (canceled)
34-39. (canceled)
(canceled)
43-44. (canceled)
47-49. (canceled)
52-62. (canceled)
Dependencies are inferred from the claim text.