公开(公告)号：EP3406730A1

公开(公告)日：2018-11-28

申请号：EP18182352.7

申请日：2011-08-29

申请人： Sirna Therapeutics, Inc.

发明人： Colletti, Steven L. ,  Gosselin, Francis ,  Jadhav, Vasant R. ,  Shaw, Anthony W. ,  Tellers, David M. ,  Tucker, Thomas J. ,  Yuan, Yu ,  Zewge, Daniel

IPC分类号： C12P21/06

CPC分类号： C12N15/113 ,  A61K47/64 ,  C12N15/111 ,  C12N15/87 ,  C12N2310/14 ,  C12N2310/3513 ,  C12N2310/3515 ,  C12N2320/32

摘要： In an embodiment the instant invention discloses a modular composition comprising 1) an oligonucleotide; 2) one or more linkers, which may be the same or different, selected from Table 1, wherein the linkers are attached to the oligonucleotide at the 2'-position of the ribose rings and/or the terminal 3'- and/or 5'-positions of the oligonucleotide; 3) optionally, one or more peptides, which may be the same or different, selected from SEQ ID NOs: 1-59, wherein the peptides are attached to the linkers; and optionally one or more lipids, solubilizing groups and/or targeting ligands attached to the oligonucleotide.

公开(公告)号：EP3366772A1

公开(公告)日：2018-08-29

申请号：EP17001972.3

申请日：2014-02-20

申请人： Sirna Therapeutics, Inc.

发明人： Chang, Wonsuk ,  Guidry, Erin, N. ,  Stanton, Matthew, G. ,  Zewge, Daniel

IPC分类号： C12N15/11 ,  C07H21/04 ,  C07H21/02 ,  C07H21/00

CPC分类号： C12N15/113 ,  C07H19/10 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/319

摘要： The present invention relates to RNAi molecules, and compositions thereof, comprising a 2' internucleoside linkage connecting the nucleotide at position 1 and the nucleotide at position 2 at the 5' end of the antisense strand. Specifically, the invention relates to single- and double-stranded short interfering nucleic acid (siNA) molecules that are capable of mediating RNA interference comprising 5' modified nucleotides that comprise, among other potential modifications, a 2' internucleoside linkage. The invention further relates to 5' modified nucleotides used as reagents to generate the RNAi molecules of the invention and methods of using the disclosed RNAi molecules.

公开(公告)号：EP3357499A1

公开(公告)日：2018-08-08

申请号：EP17001941.8

申请日：2013-12-18

申请人： Sirna Therapeutics, Inc.

发明人： Zewge, Daniel ,  Copeland, Gregory, T. ,  LI, Zhen ,  Armstrong, Joseph, D., III

IPC分类号： A61K31/712 ,  C07H21/02

CPC分类号： C12N15/113 ,  A61K31/712 ,  C07H21/02 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2330/30

摘要： This invention relates to the post-synthetic chemical modifications of RNA at the 2' - position on the ribose rings via orthogonal chemistry involving amidation reactions plus metal catalyzed alkyne-azide cycloaddition (click) reactions. RNA interference (RNAi) is an evolutionarily conserved cellular mechanism of post-transcriptional gene silencing found in fungi, plants and animals that uses small RNA molecules to inhibit gene expression in a sequence-specific manner. The RNAi machinery can be harnessed to destruct any mRNA of a known sequence.