| 发明名称 | ENA nucleic acid pharmaceuticals capable of modifying splicing of mRNA precursors | ||
| 摘要 | Oligonucleotides having a nucleotide sequence complementary to nucleotide numbers such as 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601 or 2575-2592 of the dystrophin cDNA (Gene Bank accession No. NM_004006.1) and therapeutic agents for muscular dystrophy comprising such oligonucleotides. | ||
| 申请公布号 | US9243026(B2) | 申请公布日期 | 2016.01.26 |
| 申请号 | US201414258663 | 申请日期 | 2014.04.22 |
| 申请人 | DAIICHI SANKYO COMPANY, LIMITED;Orphan Disease Treatment Institute Co., Ltd. | 发明人 | Matsuo Masafumi;Takeshima Yasuhiro;Koizumi Makoto |
| 分类号 | C07H21/04;C12N15/113;A61K38/00;A61K48/00 | 主分类号 | C07H21/04 |
| 代理机构 | Oblon, McClelland, Maier & Neustadt, L.L.P. | 代理人 | Oblon, McClelland, Maier & Neustadt, L.L.P. |
| 主权项 | 1. An oligonucleotide having the nucleotide sequence as shown in any one of SEQ ID NOS: 19, 20, 30, 41, 64, 66, or 87 in the SEQUENCE LISTING, or a pharmacologically acceptable salt thereof, wherein at least one sugar constituting the oligonucleotide is modified and/or at least one phosphate constituting the oligonucleotide is modified. | ||
| 地址 | Tokyo JP | ||