公开(公告)号：US20230113556A1

公开(公告)日：2023-04-13

申请号：US17907323

申请日：2021-03-26

申请人： NATIONAL CEREBRAL AND CARDIOVASCULAR CENTER ,  OSAKA UNIVERSITY

发明人： Mariko SHIBA ,  Tsuyoshi YAMAMOTO ,  Fumito WADA ,  Tadayuki KOBAYASHI ,  Keisuke TACHIBANA ,  Satoshi OBIKA

IPC分类号： C12N15/113 ,  A61P3/06

摘要： The present invention provides an antisense oligomer having the base sequence depicted in SEQ ID NO: 26, an antisense oligomer having a base sequence resulting from substitution, deletion, insertion, or addition of 1 to 6 bases in the base sequence depicted in SEQ ID NO: 26, a pharmaceutically acceptable salt thereof, or a pharmaceutically acceptable hydrate thereof, an oligonucleotide conjugate in which the antisense oligomer is bound with a molecule capable of binding to an asialoglycoprotein receptor, and a pharmaceutical composition containing the same.