公开(公告)号：US09133455B2

公开(公告)日：2015-09-15

申请号：US14245557

申请日：2014-04-04

申请人： Santaris Pharma A/S

发明人： Joacim Elmén ,  Phil Kearney ,  Sakari Kauppinen

IPC分类号： C07H21/04 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/113 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3515 ,  C12N2310/3521 ,  C12N2310/3533

摘要： The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 26 nucleobases which are complementary to human microRNAs selected from the group consisting of miR19b, miR21, miR122a, miR155 and miR375. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogs into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC.

摘要翻译： 本发明提供包含长度为8至26个核碱基的短单链寡核苷酸的药物组合物，其与选自miR19b，miR21，miR122a，miR155和miR375的人微小RNA互补。 短的寡核苷酸在减轻体内抑制miRNA方面特别有效。 发现将高亲和力核苷酸类似物并入寡核苷酸导致高效抗微小RNA分子，其似乎通过与miRNA靶标形成几乎不可逆的双链体而不是基于RNA切割的机制，例如与 RNaseH或RISC。

公开(公告)号：US20150299699A1

公开(公告)日：2015-10-22

申请号：US14527115

申请日：2014-10-29

申请人： Santaris Pharma A/S

发明人： Susanna OBAD ,  Sakari Kauppinen ,  Joacim Elmén ,  Morten Lindow ,  Markus Heidenblad

IPC分类号： C12N15/113 ,  A61K45/06 ,  A61K31/713

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/113 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2320/31 ,  Y02A50/465

摘要： The present invention relates to very short heavily modified oligonucleotides which target and inhibit microRNAs in vivo, and their use in medicaments and pharmaceutical compositions.