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公开(公告)号：US20160193354A1
公开(公告)日：2016-07-07
申请号：US14911264
申请日：2014-09-09
申请人： University of Vienna
IPC分类号： A61K47/48 , A61K45/06 , C12N15/113 , A61K31/713
CPC分类号： A61K47/6455 , A61K31/7088 , A61K31/713 , A61K33/24 , A61K45/06 , A61K47/60 , A61K47/645 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/351 , C12N2320/30 , A61K2300/00
摘要： The invention relates to an antisense or siRNA oligonucleotide or conjugate with improved pharmacokinetic properties, methods of producing the same as well as the use of such compounds and conjugates, e.g. as pharmaceutical composition, a pharmaceutical kit, a medicament or a tool in biomedical research.The conjugates of the invention have the formula I being P-(L-S-S-Y-X)n, wherein P represents a natural, artificial and/or modified oligonucleotide, L represents a linker group attached to one or more nucleosides at any position within the oligonucleotides sequence; S represents sulfur; X represents a ligand; Y represents a spacer and n is an integer ranging from 1 to the oligonucleotide length of P.The oligonucleotides of the invention have the formula II being P-(L-S-R)n, wherein P represents a natural, artificial and/or modified oligonucleotide, L represents a linker group attached to one or more nucleosides at any position within the oligonucleotides sequence, S represents sulfur, R represents either hydrogen or a thiol protecting group, preferably trityl or tertiary butyl, and n is an integer ranging from 1 to the oligonucleotide length of P.In addition, the pharmaceutical composition or pharmaceutical kit or conjugate can be used for the treatment of a disease or disorder that can be at least in part alleviated by therapy, wherein the disease or disorder is selected from the group consisting of bacterial infections, viral infections, cancer, metabolic diseases and immunological disorders and preferably cancer.
摘要翻译： 本发明涉及具有改善的药代动力学性质的反义或siRNA寡核苷酸或缀合物，其制备方法以及这些化合物和缀合物的使用，例如， 作为药物组合物，药物试剂盒，药物或生物医学研究中的工具。 本发明的缀合物具有式I是P-（L-S-S-Y-X）n，其中P表示天然的人造和/或修饰的寡核苷酸，L表示在寡核苷酸序列内的任何位置连接至一个或多个核苷的连接基团; S代表硫; X表示配体; Y表示间隔基，n是从1到寡核苷酸长度P的整数。本发明的寡核苷酸具有式II为P-（LSR）n，其中P表示天然的，人造的和/或修饰的寡核苷酸，L 表示在寡核苷酸序列内的任何位置连接至一个或多个核苷的连接基团，S表示硫，R表示氢或硫醇保护基团，优选三苯甲基或叔丁基，n为1至寡核苷酸长度的整数 另外，药物组合物或药物试剂盒或缀合物可用于治疗可以至少部分地通过治疗减轻的疾病或病症，其中所述疾病或病症选自细菌感染 ，病毒感染，癌症，代谢疾病和免疫学疾病，优选癌症。
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公开(公告)号：US09968686B2
公开(公告)日：2018-05-15
申请号：US14911264
申请日：2014-09-09
申请人： University of Vienna
IPC分类号： C07H21/04 , A61K47/48 , A61K31/7088 , A61K33/24 , A61K45/06 , A61K31/713 , C12N15/113
CPC分类号： A61K47/6455 , A61K31/7088 , A61K31/713 , A61K33/24 , A61K45/06 , A61K47/60 , A61K47/645 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/351 , C12N2320/30 , A61K2300/00
摘要： The invention relates to an antisense or siRNA oligonucleotide or conjugate with improved pharmacokinetic properties, methods of producing the same as well as the use of such compounds and conjugates, e.g. as pharmaceutical composition, a pharmaceutical kit, a medicament or a tool in biomedical research.The conjugates of the invention have the formula I being P-(L-S—S—Y—X)n, wherein P represents a natural, artificial and/or modified oligonucleotide, L represents a linker group attached to one or more nucleosides at any position within the oligonucleotides sequence; S represents sulfur; X represents a ligand; Y represents a spacer and n is an integer ranging from 1 to the oligonucleotide length of P.The oligonucleotides of the invention have the formula II being P-(L-S—R)n, wherein P represents a natural, artificial and/or modified oligonucleotide, L represents a linker group attached to one or more nucleosides at any position within the oligonucleotides sequence, S represents sulfur, R represents either hydrogen or a thiol protecting group, preferably trityl or tertiary butyl, and n is an integer ranging from 1 to the oligonucleotide length of P.In addition, the pharmaceutical composition or pharmaceutical kit or conjugate can be used for the treatment of a disease or disorder that can be at least in part alleviated by therapy, wherein the disease or disorder is selected from the group consisting of bacterial infections, viral infections, cancer, metabolic diseases and immunological disorders and preferably cancer.
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