公开(公告)号：US20240060068A1

公开(公告)日：2024-02-22

申请号：US18256428

申请日：2021-12-10

申请人： Francis G. FANG ,  Dai-Shik KIM ,  Hyeong Wook CHOI ,  Yoshinori TAKAHASHI ,  Kenji KIKUTA ,  Hikaru KAWASHIMA ,  Toshiki KUROKAWA ,  Tamaki HOSHIKAWA ,  Mingde SHAN ,  John WANG ,  Eisai R&D Management Co., Ltd.

发明人： Francis G. Fang ,  Dae-Shik Kim ,  Hyeong Wook Choi ,  Yoshinori Takahashi ,  Kenji Kikuta ,  Hikaru Kawashima ,  Wataru Itano ,  Toshiki Kurokawa ,  Tamaki Hoshikawa ,  Mingde SHAN ,  John Wang

IPC分类号： C12N15/113 ,  C07H21/04

CPC分类号： C12N15/113 ,  C07H21/04 ,  C12N2310/341 ,  C12N2310/3233 ,  C12N2310/315

摘要： Gapmers or pharmaceutically acceptable salt of the gapmers and methods of making the gapmers are provided. The gapmers include a gap region that contains deoxyribonucleosides linked to each other by phosphorothioate bonds, a 5′ wing region positioned at the 5′ end of the gap region that contains morpholino monomers linked to each other by phosphorodiamidate bonds, and a 3′ wing region positioned at the 3′ end of the gap region that contains morpholino monomers linked to each other by phosphorodiamidate bonds. Antisense oligonucleotides are also provided. These antisense oligonucleotides are useful in the preparation of gapmers for inhibition of Tau mRNA transcription.