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US 2023/0416748 A1Published application
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsFrederic Tremblay, James D. McIninch
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Abstract
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disease in a subject.
Claims38
A double stranded ribonucleic acid (dsRNA) for inhibiting expression of ketohexokinase (KHK) in a cell,
- (a) wherein said dsRNA comprises a sense strand and an antisense strand forming a double stranded region, wherein the antisense strand comprises a region of complementarity to an mRNA encoding KHK, and wherein the region of complementarity comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the antisense nucleotide sequences in any one of Tables 2-5;
- or
- (b) wherein said dsRNA comprises a sense strand and an antisense strand forming a double stranded region, wherein the sense strand comprises at least 15 contiguous nucleotides differing by no more than three nucleotides from any one of the nucleotide sequence of nucleotides 943-965;
- 788-810;
- 734-756;
- 1016-1038;
- 1013-1035;
- 1207-1229;
- 1149-1171;
- 574-596;
- 1207-1229 or 828-850 of SEQ ID NO:
- 1, and the antisense strand comprises at least 15 contiguous nucleotides from the corresponding nucleotide sequence of SEQ ID NO:2.
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Dependencies are inferred from the claim text.