首页> 外国专利> RNAI AGENTS FOR INHIBITING EXPRESSION OF 17BETA-HSD TYPE 13- (HSD17B13), COMPOSITIONS THEREOF, AND METHODS OF USE

RNAI AGENTS FOR INHIBITING EXPRESSION OF 17BETA-HSD TYPE 13- (HSD17B13), COMPOSITIONS THEREOF, AND METHODS OF USE

机译:用于抑制17beta-HSD型13-(HSD17B13)的表达的RNAi剂,其组合物和使用方法

摘要

The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit l7P-hydroxy steroid dehydrogenase type 13 (HSD17B13 or 17β-Η8013) 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. Delivery7 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.
机译:本公开涉及RNAi试剂,例如双链RNAi剂,能够抑制L7P-羟基类固醇脱氢酶型13(HSD17B13或17β-η8013)基因表达。还公开了包含HSD17B13 RNAi剂及其使用方法的药物组合物。本文公开的HSD17B13 RNAi试剂可以与靶向配体缀合,以促进递送给细胞,包括肝细胞。体内HSD17B13 RNAi试剂的递送提供了HSD17B13基因表达的抑制。 RNAi剂可用于治疗HSD17B13相关疾病和疾病的方法,包括非酒精性脂肪肝疾病(NAFLD),非酒精脂肪疏皮性(NASH),肝纤维化和酗酒或非酒精肝病,包括肝硬化。

著录项

  • 公开/公告号EP3852769A1

    专利类型

  • 公开/公告日2021-07-28

    原文格式PDF

  • 申请/专利权人 ARROWHEAD PHARMACEUTICALS INC.;

    申请/专利号EP20190861799

  • 发明设计人 LI ZHEN;ZHU RUI;MORALES SHAWN A.;

    申请日2019-09-18

  • 分类号A61K31/7105;A61K31/713;C12N15/113;

  • 国家 EP

  • 入库时间 2022-08-24 20:12:55

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