LI, Zhen,ZHU, Rui,PEI, Tao,KANNER, Steven,WONG, So
申请号:
AU2018329190
公开号:
AU2018329190A1
申请日:
2018.09.10
申请国别(地区):
AU
年份:
2020
代理人:
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