发明申请
WO2019051402A1 RNAI AGENTS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)
审中-公开
基本信息:
- 专利标题： RNAI AGENTS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)
- 申请号：PCT/US2018/050248 申请日：2018-09-10
- 公开(公告)号：WO2019051402A1 公开(公告)日：2019-03-14
- 发明人： LI, Zhen , ZHU, Rui , PEI, Tao , KANNER, Steven , WONG, So
- 申请人： ARROWHEAD PHARMACEUTICALS, INC.
- 申请人地址： 225 South Lake Avenue, Suite 1050 Pasadena, CA 91101 US
- 专利权人： ARROWHEAD PHARMACEUTICALS, INC.
- 当前专利权人： ARROWHEAD PHARMACEUTICALS, INC.
- 当前专利权人地址： 225 South Lake Avenue, Suite 1050 Pasadena, CA 91101 US
- 代理机构： TEIGEN, Robert, Michael
- 优先权： US62/556,818 20170911; US62/643,927 20180316; US62/720,434 20180821
- 主分类号： A61K31/7125
- IPC分类号： A61K31/7125
摘要：
The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, capable of inhibiting Apolipoprotein C-III (also called APOC3, apoC-III, APOC-III, and APO C- III) gene expression, and compositions that include APOC3 RNAi agents. The APOC3 RNAi agents disclosed herein may be conjugated to targeting ligands. including ligands that include N -acetylgalactosamine, to facilitate the delivery to cells, including to hepatocytes. Pharmaceutical compositions that include one or more APOC3 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the APOC3 RNAi agents in vivo provides for inhibition of APOC3 gene expression, and can result in lower triglycerides and or cholesterol levels in the subject. The APOC3 RNAi agents can be used in methods of treatment of APOC3-related diseases and disorders, including hypertriglyceridemia, cardiovascular disease, and other metabolic-related disorders and diseases.
IPC结构图谱:
| A | 人类生活必需 |
| --A61 | 医学或兽医学；卫生学 |
| ----A61K | 医用、牙科用或梳妆用的配制品 |
| ------A61K31/00 | 含有机有效成分的医药配制品 |
| --------A61K31/70 | .碳水化合物；糖；其衍生物 |
| ----------A61K31/7088 | ..含有3个或更多个核苷或核苷酸的化合物 |
| ------------A61K31/7125 | ...含有修饰核苷酸间键合，即非3′,5′磷酸二酯键的核酸或寡核苷酸 |