公开(公告)号：EP2825890A1

公开(公告)日：2015-01-21

申请号：EP13714874.8

申请日：2013-03-18

申请人： Max-Delbrück-Centrum für Molekulare Medizin

发明人： LANDTHALER, Markus ,  MUNSCHAUER, Mathias ,  BALTZ, Alexander

IPC分类号： G01N33/58 ,  C12Q1/68 ,  C12N15/113

CPC分类号： C12Q1/6874 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/13 ,  C12N2310/333 ,  C12N2310/335 ,  C12N2320/11 ,  C12Q1/6804 ,  C12Q1/6806 ,  C12Q1/6827 ,  C12Q2535/101 ,  C12Q2600/136 ,  G01N33/6842 ,  C12Q2521/327 ,  C12Q2521/537 ,  C12Q2523/101 ,  C12Q2523/313 ,  C12Q2525/173 ,  C12Q2527/137 ,  C12Q2563/149 ,  C12Q2565/125 ,  C12Q2527/125

摘要： The invention relates to an in vitro method for identifying the sequence of one or more poly(A)+RNA molecules that physically interacts with protein. The present invention provides a method to define the protein-bound transcriptome under any given cellular condition, such as a disease condition or after treatment with any given substance, drug, or other cellular perturbation. The invention also relates to a method for identification of a drug target and a method for the identification of one or more biomarkers, preferably for identification of a panel of biomarkers, for any given medical condition, comprising the method of the invention.

公开(公告)号：EP2699269A1

公开(公告)日：2014-02-26

申请号：EP12720671.2

申请日：2012-04-23

申请人： Prosensa Technologies B.V.

发明人： AGUILERA DIEZ, Maria Begoña ,  DE VISSER, Peter Christian ,  MULDERS, Susan Allegonda Maria

IPC分类号： A61K47/48 ,  A61P21/00 ,  C12N15/113

CPC分类号： C12N15/1137 ,  A61K31/7088 ,  A61K47/549 ,  A61K47/62 ,  A61K47/64 ,  C07K19/00 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/333 ,  C12N2310/3341 ,  C12N2310/346 ,  C12N2310/3513 ,  C12N2320/32 ,  C12Y207/11001 ,  C12N2310/3521

摘要： The current invention provides new compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1), spino-cerebellar ataxia 8 and/or Huntington's disease-like 2 caused by expansions of CUG repeats in the transcripts of DM1/DMPK, SCA8 or JPH3 genes.

摘要翻译： 当前发明提供了治疗，延缓和/或预防人类遗传病症的新化合物：如强直性肌营养不良1型（DM1），脊髓小脑性小脑共济失调8和/或亨廷顿氏病样2通过在转录CUG重复的膨胀引起的 的DM1 / DMPK，SCA8或JPH3基因。

公开(公告)号：EP1406667B1

公开(公告)日：2008-02-20

申请号：EP02748499.7

申请日：2002-07-08

申请人： Topigen Pharmaceuticals Inc.

发明人： RENZI, Paolo ,  ALLAM, Mustapha ,  ALLAKHVERDI, Zoulfia

IPC分类号： A61K48/00 ,  C07H19/00 ,  C07H21/00 ,  C12N15/11

CPC分类号： C12N15/113 ,  A61K31/7076 ,  A61K38/00 ,  C07H19/16 ,  C07H19/23 ,  C07H21/00 ,  C07H21/04 ,  C12N15/1138 ,  C12N2310/315 ,  C12N2310/333

摘要： The invention relates to the use of nucleotide substitutes for increasing the in vivo efficacy of nucleic acid molecules and also for inhibiting inflammation in mammals. More particularly, the present invention relates to the use of 2'6'diaminopurine (DAP) and analogs thereof per se in anti-inflammatory compositions, and also for preparing nucleic acid molecules having an increased in vivo physiological efficiency and a reduced toxicity as compared to conventional oligos. The invention is particularly useful for the preparation of antisense oligonucleotides for treating pulmonary/respiratory diseases such as cystic fibrosis, asthma, chronic bronchitis, chronic obstructive lung disease, eosinophilic bronchitis, allergies, allergic rhinitis, pulmonary fibrosis, adult respiratory distress syndrome, sinusitis, respiratory syncytial virus or other viral respiratory tract infection and cancer.

公开(公告)号：EP1496943A4

公开(公告)日：2007-05-23

申请号：EP03726231

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： A61K38/00 ,  C07H21/00 ,  C12N5/02 ,  C12N15/113 ,  A61K48/00 ,  C07H21/04

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

公开(公告)号：EP1670915A2

公开(公告)日：2006-06-21

申请号：EP04816223.4

申请日：2004-09-15

申请人： Sirna Therapeutics, Inc.

发明人： JADHAV, Vasant ,  KOSSEN, Karl ,  ZINNEN, Shawn ,  VAISH, Narendra ,  MCSWIGGEN, James

IPC分类号： C12N15/11

CPC分类号： C12N15/113 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N15/1137 ,  C12N15/1138 ,  C12N15/87 ,  C12N2310/111 ,  C12N2310/12 ,  C12N2310/121 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/316 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/333 ,  C12N2310/346 ,  C12N2310/53 ,  C12N2330/30 ,  C12Y103/01022 ,  C12N2310/3521 ,  C12N2310/3533

摘要： This invention relates to compounds, compositions, and methods useful for modulating HCVgene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of HCV gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HCV genes.

公开(公告)号：EP0549615B1

公开(公告)日：2006-02-22

申请号：EP91915355.1

申请日：1991-08-12

申请人： ISIS PHARMACEUTICALS, INC.

发明人： COOK, Philip, Dan ,  KAWASAKI, Andrew, Mamoto

IPC分类号： C07H21/00 ,  A61K31/70 ,  C07H19/16

CPC分类号： C12N15/113 ,  A61K31/70 ,  A61K38/00 ,  A61K48/00 ,  C07H19/04 ,  C07H19/06 ,  C07H19/10 ,  C07H19/16 ,  C07H19/20 ,  C07H21/00 ,  C07H23/00 ,  C07J43/003 ,  C12N9/0069 ,  C12N15/1137 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/33 ,  C12N2310/333 ,  C12N2310/336 ,  C12N2310/3521 ,  C12N2310/3523 ,  C12N2310/3527 ,  C12N2310/3531 ,  C12N2310/3533 ,  C12Q1/68 ,  C12Y113/11012

摘要： Compositions and methods are provided for the treatment and diagnosis of diseases amenable to modulation of the production of selected proteins. In accordance with preferred embodiments, oligonucleotides and oligonucleotide analogs are provided which are specifically hybridizable with a selected sequence of RNA or DNA wherein at least one of the 2'-deoxyfuranosyl moieties of the nucleoside unit is modified. Treatment of HIV, herpes virus, papillomavirus and other infections is provided.

公开(公告)号：EP1575977A2

公开(公告)日：2005-09-21

申请号：EP03814325.1

申请日：2003-12-18

申请人： Dynavax Technologies Corporation

发明人： DINA, Dino ,  FEARON, Karen, L. ,  MARSHALL, Jason

IPC分类号： C07H21/02 ,  C07H21/04 ,  C07H21/00 ,  A01N43/04

CPC分类号： C12N15/117 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/333 ,  C12N2310/336

摘要： The invention provides immunomodulatory polynucleotides and methods for immunomodulation of individuals using the immunomodulatory polynucleotides.

公开(公告)号：EP0716604B1

公开(公告)日：2005-03-16

申请号：EP94927361.9

申请日：1994-08-31

申请人： ISIS PHARMACEUTICALS, INC.

发明人： COOK, Phillip Dan ,  MANOHARAN, Muthiah

IPC分类号： A61K31/70 ,  C07H19/00 ,  C07H21/00 ,  C07J51/00 ,  C07J43/00

CPC分类号： C07H21/00 ,  A61K38/00 ,  A61K48/00 ,  C07H19/04 ,  C07H19/06 ,  C07H19/10 ,  C07H19/16 ,  C07H19/20 ,  C07H23/00 ,  C07J43/003 ,  C07J51/00 ,  C12N9/0069 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/33 ,  C12N2310/333 ,  C12N2310/336 ,  C12N2310/3521 ,  C12N2310/3523 ,  C12N2310/3527 ,  C12N2310/3531 ,  C12N2310/3533 ,  C12Q1/68 ,  C12Y113/11012

公开(公告)号：EP1502950A2

公开(公告)日：2005-02-02

申请号：EP04000538.1

申请日：1995-02-23

申请人： RIBOZYME PHARMACEUTICALS, INC.

发明人： Stinchcomb, Dan T. ,  Chowrira, Bharat ,  Direnzo, Anthony ,  Draper, Kenneth G. ,  Dudycz, Lech W. ,  Grimm, Susan ,  Karpeisky, Alexander ,  Kisich, Kevin ,  Matulic-Adamic, Jasenka ,  McSwiggen, James A. ,  Modak, Anil ,  Pavco, Pamela ,  Beigelman, Leonid ,  Sullivan, Sean M. ,  Sweedler, David ,  Thompson, James D. ,  Tracz, Danuta ,  Usman, Nassim ,  Wincott, Francine E., c/o Eyetech Pharmaceutical ,  Woolf, Tod

IPC分类号： C12N15/10 ,  C07H21/04

CPC分类号： C12N15/1003 ,  A61K38/00 ,  A61K48/00 ,  C12N15/101 ,  C12N15/113 ,  C12N15/1131 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1138 ,  C12N15/85 ,  C12N2310/111 ,  C12N2310/121 ,  C12N2310/122 ,  C12N2310/123 ,  C12N2310/1241 ,  C12N2310/126 ,  C12N2310/127 ,  C12N2310/32 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/3513 ,  C12N2310/3523 ,  C12N2310/3527 ,  C12N2310/3533 ,  C12N2310/3535 ,  C12N2760/18511 ,  C12N2830/38

摘要： Disclosed are processes for deprotecting and/or purifying chemically synthesised RNAs, especially enzymatic nucleic acids or antisense oligonucleotides, having one or more chemical modifications.

摘要翻译： 公开了用于去保护和/或纯化化学合成的RNA，特别是具有一种或多种化学修饰的酶核酸或反义寡核苷酸的方法。

公开(公告)号：EP1418179A2

公开(公告)日：2004-05-12

申请号：EP03078862.4

申请日：1991-01-11

申请人： ISIS PHARMACEUTICALS, INC.

发明人： Cook, Philip, Dan ,  Ecker, David, J. ,  Guinosso, Charles, John ,  Acevedo, Oscar, Leobardo ,  Kawasaki, Andrew, Mamoto ,  Ramasamy, Kandasamy

IPC分类号： C07H19/052 ,  C07H19/06 ,  C07H19/12 ,  C07H19/14 ,  C07H19/16 ,  C07H19/22 ,  C07H19/23 ,  A61K31/7052

CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C07H19/04 ,  C07H19/06 ,  C07H19/10 ,  C07H19/16 ,  C07H19/20 ,  C07H21/00 ,  C07H23/00 ,  C07J43/003 ,  C07K14/005 ,  C12N9/0069 ,  C12N15/1137 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/33 ,  C12N2310/333 ,  C12N2310/336 ,  C12N2310/3511 ,  C12N2310/3521 ,  C12N2310/3523 ,  C12N2310/3527 ,  C12N2310/3531 ,  C12N2310/3533 ,  C12N2740/16322 ,  C12Q1/68 ,  C12Q1/6813 ,  C12Y113/11012

摘要： Compositions and methods for modulating the activity of RNA and DNA are disclosed. In accordance with preferred embodiments, antisense compositions are prepared comprising targeting and reactive portions. Reactive portions which act, alternatively, through phosphorodiester bond cleavage, through backbone sugar bond cleavage or through base modification are preferably employed. Groups which improve the pharmacodynamic and pharmacokinetic properties of the oligonucleotides are also useful in accordance with certain embodiments of this invention. Delivery of the reactive or non-reactive functionalities into the minor groove formed by the hybridization of the composition with the target RNA is also preferably accomplished. Therapeutics, diagnostics and research methods are also disclosed. Synthetic nucleosides and nucleoside fragments are also provided useful for elaboration of oligonucleotides and oligonucleotide analogs for such purposes.

摘要翻译： 组合物和方法用于调节RNA和DNA的活性的游离缺失光盘。 结合优选实施例雅舞蹈，反义的组合物制备包含靶向和反应性的部分。 哪通过骨干糖或通过碱基修饰作用，可选地，通过磷酸二酯键裂解，键裂解反应性部分优选地使用。 其改善了寡核苷酸的药效学和药代动力学性质的基团是在舞蹈雅与本发明的某些实施方案因此是有用的。 因此，反应性或非反应性官能团的递送入由与所述目标RNA的组合物的杂交所形成的小沟，优选完成的。 因此，治疗，诊断和研究方法是游离缺失盘。 合成核苷和核苷片段从而为寡核苷酸和寡核苷酸类似物的阐述用于搜索目的提供了有用的。