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AssigneeArrowhead Pharmaceuticals, Inc.
InventorsZhen Li, Rui Zhu, Shawn A Morales
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The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit 17β-hydroxysteroid dehydrogenase type 13 (HSD17B13 or 17β-HSD13) gene expression. Also disclosed are pharmaceutical compositions that include HSD17B13 RNAi agents and methods of use thereof. The HSD17B13 RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Delivery of the HSD17B13 RNAi agents in vivo provides for inhibition of HSD17B13 gene expression. The RNAi agents can be used in methods of treatment of HSD17B13-related diseases and disorders, including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), hepatic fibrosis, and alcoholic or non-alcoholic liver diseases, including cirrhosis.
An RNAi agent for inhibiting expression of an HSD17B13 gene, comprising:
An RNAi agent comprising a chemical structure as follows with the antisense strand (5′→3′) SEQ ID NO:4 and the sense strand (5′→3′) SEQ ID NO:15:
An RNAi agent comprising a chemical structure as follows with the antisense strand (5′→3′) SEQ ID NO:4 and the sense strand (5′→3′) SEQ ID NO:
An RNAi agent comprising 1) an antisense strand that comprises the structure of (5′→3′) usCfsasUfcUfaucagAfcUfuCfuUfaCfsg (SEQ ID NO:4), and 2) a sense strand that comprises the structure of (5′→3′) (NAG37)s(invAb)scguaagaaGfuCfuGfauagaugas(invAb) (SEQ ID NO:15), wherein:
Dependencies are inferred from the claim text.