专利名称 | COMPOSITIONS AND METHODS | ||
申请号 | RU2015151204 | 申请日 | |
公开(公告)号 | RU2686080C2 | 公开(公告)日 | |
申请(专利权)人 | IONIS PHARMACEUTICALS INC | 发明人 | PRAKASH Thazha P; SETH Punit P; SWAYZE Eric E |
专利来源 | 国家知识产权局 | 转化方式 | |
摘要 |
FIELD : pharmaceuticals. SUBSTANCE : invention refers to compounds for treating metabolic diseases and can be used in medicine. Disclosed is a compound comprising a single-chain modified oligonucleotide and a conjugating group, or a pharmaceutically acceptable salt thereof, wherein the conjugating group contains , where the cleavable fragment (CM) is a bond or a group which can be split under physiological conditions; modified oligonucleotide has formula : Ges Ges Tes Tds mCds mCds mCds Gds Ads Gds Gds Tds Gds mCes mCes mCes Ae, where A = adenine, mC = 5' -methylcytosine, G = guanine, T = thymine, e = 2' -O-methoxyethyl modified nucleoside, d = 2' -deoxynucleoside and s = thiophosphate internucleoside linkage; and the conjugating group is bound to the modified oligonucleotide by 5' -end of modified oligonucleotide. EFFECT : novel compounds and compositions based thereon for treating metabolic diseases are disclosed. 5 cl, 113 ex, 108 tbl, list of sequences |