- 专利标题： NUCLEIC ACID COMPLEX
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申请号： US17760908申请日： 2020-09-16
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公开(公告)号： US20220370491A1公开(公告)日： 2022-11-24
- 发明人: Takanori Yokota , Testuya Nagata , Hideki Furukawa , Takatoshi Yogo , Yasuo Nakagawa , Shigekazu Sasaki , Ryosuke Tokunoh , Tomohiro Seki , Kosuke Hidaka , Fumiaki Kikuchi , Osamu Kubo , Takahito Kasahara , Takuto Kojima , Junsi Wang , Norihito Tokunaga
- 申请人： National University Corporation Tokyo Medical and Dental University , Takeda Pharmaceutical Company Limited
- 申请人地址： JP Tokyo,; JP Chuo-ku, Osaka-shi, Osaka
- 专利权人： National University Corporation Tokyo Medical and Dental University,Takeda Pharmaceutical Company Limited
- 当前专利权人： National University Corporation Tokyo Medical and Dental University,Takeda Pharmaceutical Company Limited
- 当前专利权人地址： JP Tokyo,; JP Chuo-ku, Osaka-shi, Osaka
- 优先权： JP2019-168930 20190918
- 国际申请： PCT/JP2020/035117 WO 20200916
- 主分类号： A61K31/713
- IPC分类号： A61K31/713 ; A61K47/54 ; A61P25/00 ; A61K9/00
摘要：
The object of the present invention is to provide a nucleic acid agent that is efficiently delivered to the nervous system, for example, the central nervous system to which drug delivery can be prevented by BBB, and produces an antisense effect on the target transcriptional product at the delivery site, and a composition comprising the same. In an embodiment, the present invention provides a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand that hybridizes to a part of a target transcriptional product and has an antisense effect on the target transcriptional product, and a second nucleic acid strand that comprises a base sequence complementary to the first nucleic acid strand and is bound to a C22-35 alkyl group optionally substituted with a hydroxy group or an analog thereof.