公开(公告)号：EP2606057A2

公开(公告)日：2013-06-26

申请号：EP11721849.5

申请日：2011-04-26

申请人： Isis Pharmaceuticals, Inc. ,  Alnylam Pharmaceuticals, Inc.

发明人： PRAKASH, Thazha, P. ,  SETH, Punit, P. ,  MANORHARAN, Muthiah ,  SWAYZE, Eric, E. ,  RAJEEV, Kallanthottathil, G. ,  ZLATEV, Ivan

IPC分类号： C07H19/10

CPC分类号： C07H19/10 ,  C12N15/113

摘要： Provided herein are modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom. More particularly, modified 5' diphosphate nucleosides are provided that can be further modified at the 2' and 5' positions. In some embodiments, the oligomeric compounds provided herein are expected to hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The oligomeric compounds are also expected to be useful as primers and probes in diagnostic applications.

公开(公告)号：EP2580228A1

公开(公告)日：2013-04-17

申请号：EP11726017.4

申请日：2011-06-02

申请人： Isis Pharmaceuticals, Inc.

发明人： SETH, Punit, P. ,  PRAKASH, Thazha, P. ,  SWAYZE, Eric, E.

IPC分类号： C07H19/073 ,  C07H21/04 ,  C07H19/173 ,  C12N15/113

CPC分类号： C07H19/06 ,  C07H19/073 ,  C07H19/173 ,  C07H21/00 ,  C07H21/04 ,  C12N15/113 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341

摘要： Provided herein are 2'-amino and 2'-thio bicyclic nucleosides and oligomenc compounds prepared therefrom. The novel bicyclic nucleosides provided herein are expected to be useful for enhancing one or more properties of the oligomeric compounds they are incorporated into such as nuclease resistance.

公开(公告)号：EP2358398A2

公开(公告)日：2011-08-24

申请号：EP09744287.5

申请日：2009-10-23

申请人： Isis Pharmaceuticals, Inc.

发明人： SWAYZE, Eric, E. ,  BHAT, Balkrishen ,  LIMA, Walter, F. ,  PRAKASH, Thazha, P. ,  KINBERGER, Garth, A.

IPC分类号： A61K48/00

CPC分类号： C12N15/1137 ,  C07H21/00 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/351

摘要： The present invention provides oligomeric compounds and uses thereof. In certain embodiments, such oligomeric compounds are useful as antisense compounds. Certain such antisense compounds are useful as RNase H antisense compounds or as RNAi compounds.

公开(公告)号：EP2358397A2

公开(公告)日：2011-08-24

申请号：EP09744015.0

申请日：2009-10-23

申请人： Isis Pharmaceuticals, Inc.

发明人： PRAKASH, Thazha, P. ,  SWAYZE, Eric, E.

IPC分类号： A61K48/00

CPC分类号： C07H19/10 ,  A61K31/00 ,  C07H21/00

摘要： The present invention provides modified nucleosides and oligomeric compounds prepared therefrom. More particularly, the present invention provides modified nucleosides having at least one 5′-substituent and a 2′-O-substituent, oligomeric compounds comprising at least one of these modified nucleosides and methods of using the oligomeric compounds. In some embodiments, the oligomeric compounds provided herein are expected to hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.

摘要翻译： 本发明提供了经修饰的核苷和由其制备的低聚化合物。 更具体地，本发明提供了修饰核苷具有至少一个5“取代基，2'-O-取代的，低聚的化合物包括合成修饰的核苷以及使用所述寡聚化合物的方法中的至少一个。 在一些实施方案中，预期在所提供的低聚化合物杂交至靶RNA中的靶RNA的正常功能的丢失而导致的部分。

公开(公告)号：EP1766071A2

公开(公告)日：2007-03-28

申请号：EP05757763.7

申请日：2005-06-02

申请人： ISIS PHARMACEUTICALS, INC.

发明人： BHAT, Balkrishen ,  PRAKASH, Thazha, P. ,  DANDE, Prasad ,  ALLERSON, Charles ,  GRIFFEY, Richard ,  SWAYZE, Eric E.

IPC分类号： C12Q1/68 ,  C07H21/02 ,  A61K48/00

CPC分类号： C12N15/113 ,  C07H21/02 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2320/51 ,  C12N2310/3521

摘要： The present invention provides douse stranded compositions wherein each strand is modified to have a motif defined by positioning of ß-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif or an alternating motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In some embodiments, the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

公开(公告)号：EP1766052A1

公开(公告)日：2007-03-28

申请号：EP04754188.3

申请日：2004-06-03

申请人： ISIS PHARMACEUTICALS, INC.

发明人： BHAT, Balkrishen ,  SWAYZE, Eric ,  ALLERSON, Charles ,  DANDE, Prasad ,  PRAKASH, Thazha, P. ,  GRIFFEY, Richard, H.

IPC分类号： C12Q1/68 ,  A01N43/04 ,  C07H21/04 ,  A61K31/07

CPC分类号： C12N15/113 ,  A61K31/07 ,  C07H21/04 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/341 ,  C12N2320/51 ,  C12N2310/3521

摘要： The present invention provides double stranded compositions wherein the first strand comprises three different regions and the second strand is native RNA. Each region has native or modified ribofuranosyl sugar moieties that are different than those of the other two regions. At least a portion of the first oligomeric compound is complementary to and hybridizes to a nucleic acid target. The present invention also provides methods for modulating gene expression using the modified oligomeric compounds and compositions of oligomeric compounds.

公开(公告)号：EP1496943A2

公开(公告)日：2005-01-19

申请号：EP03726231.8

申请日：2003-04-09

申请人： ISIS PHARMACEUTICALS, INC.

发明人： RAVIKUMAR, Vasulinga ,  PRAKASH, Thazha, P. ,  BHAT, Balkrishen

IPC分类号： A61K48/00 ,  C07H21/04

CPC分类号： C07H21/00 ,  A61K38/00 ,  C12N15/113 ,  C12N2310/315 ,  C12N2310/33 ,  C12N2310/333 ,  C12N2310/3341 ,  C12N2310/341

摘要： Oligomeric compounds having at least one phosphorothioate monoester are provided having increased nuclease resistance and binding affinity to a complementary strand of nucleic acid. Such oligomeric compounds are useful for diagnostics and other research purposes, for modulating the expression of a protein in organisms, and for the diagnosis, detection and treatment of other conditions responsive to oligonucleotide therapeutics.

公开(公告)号：EP1102783A1

公开(公告)日：2001-05-30

申请号：EP99943665.2

申请日：1999-08-09

申请人： ISIS PHARMACEUTICALS, INC.

发明人： MANOHARAN, Muthiah ,  COOK, Phillip, Dan ,  PRAKASH, Thazha, P. ,  KAWASAKI, Andrew, M.

IPC分类号： C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C07H19/06 ,  C07H19/16 ,  C07H19/10 ,  C07H19/20

CPC分类号： C07H21/00 ,  C07H15/203 ,  C07H19/04 ,  C07H19/06 ,  C07H19/16 ,  Y02P20/582

摘要： Nucleosidic monomers and oligomeric compounds prepared therefrom are provided which have increased nuclease resistance, substituent groups (such as 2'-aminooxy groups) for increasing binding affinity to complementary strand, and regions of 2'-deoxy-erythro-pentofuranosyl nucleotides that activate RNase H. Such oligomeric compounds are useful for diagnostics and other research purposes, for modulating the expression of a protein in organisms, and for the diagnosis, detection and treatment of other conditions susceptible to oligonucleotide therapeutics.

公开(公告)号：EP2751269A1

公开(公告)日：2014-07-09

申请号：EP12827475.0

申请日：2012-08-29

申请人： Isis Pharmaceuticals, Inc. ,  Board of Regents of the University of Texas System

发明人： PRAKASH, Thazha, P. ,  SWAYZE, Eric, E. ,  COREY, David ,  YU, Dongbo

IPC分类号： C12N15/11 ,  C07H21/04 ,  C12N5/00

CPC分类号： C12N15/113 ,  A61K31/7088 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/343 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2310/533 ,  C12N2320/34

摘要： Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules.

公开(公告)号：EP2742136A1

公开(公告)日：2014-06-18

申请号：EP12751399.2

申请日：2012-08-08

申请人： Isis Pharmaceuticals, Inc.

发明人： OESTERGAARD, Michael ,  PRAKASH, Thazha, P. ,  SETH, Punit, P. ,  SWAYZE, Eric, E.

IPC分类号： C12N15/113 ,  A61K31/712

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/312 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/316 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2320/34 ,  C12N2310/3525

摘要： The present invention provides gapped oligomeric compounds comprising at least one 5'- substituted P-D-2'-deoxyribonucleoside in the gap region. Certain such gapped oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited to, nucleic acids in a cell. The oligomeric compounds provided herein have improved properties such as selectivity, potency and improved proinflammatory profile. In certain embodiments, hybridization results in modulation of the amount of activity or expression of the target nucleic acid in a cell.

摘要翻译： 本发明提供了有效的低聚化合物。 更具体地，本文提供的有间隙的寡聚化合物在间隙区域中包含至少一个修饰的核苷间键。 这种有效的低聚化合物具有一种或多种改进的性质，例如选择性，效力，改善的毒性特征和/或改善的促炎症状。 某些这样的寡聚化合物可用于与互补核酸杂交，包括但不限于细胞中的核酸。 在某些实施方案中，杂交导致调节细胞中靶核酸的量活性或表达。