公开(公告)号：EP0731807B1

公开(公告)日：2004-03-17

申请号：EP95902698.0

申请日：1994-11-29

申请人： ISIS PHARMACEUTICALS, INC.

发明人： COOK, Phillip, Dan ,  RAMASAMY, Kanda, S. ,  MANOHARAN, Muthiah

IPC分类号： C07H19/16 ,  C07H19/167 ,  C07H19/173

CPC分类号： C07H19/16 ,  A61K38/00 ,  A61K47/51 ,  A61K48/00 ,  C07H19/04 ,  C07H19/06 ,  C07H19/10 ,  C07H19/20 ,  C07H21/00 ,  C07H23/00 ,  C07J43/003 ,  C12N9/0069 ,  C12N15/113 ,  C12N15/1131 ,  C12N15/1132 ,  C12N15/1133 ,  C12N15/1135 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/3125 ,  C12N2310/315 ,  C12N2310/318 ,  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： This invention presents novel purine-based compounds for inclusion into oligonucleotides. The compounds of the invention, when incorporated into oligonucleotides are especially useful as 'antisense' agents -- agents that are capable of specific hybridization with a nucleotide sequence of an RNA. Oligonucleotides are used for a variety of therapeutic and diagnostic purposes, such as treating diseases, regulating gene expression in experimental systems, assaying for RNA and for RNA products through the employment of antisense interactions with such RNA, diagnosing diseases, modulating the production of proteins, and cleaving RNA in site specific fashions. The compounds of the invention include novel heterocyclic bases, nucleosides, and nucleotides. When incorporated into oligonucleotides, the compounds of the invention can be useful for modulating the activity of RNA.

公开(公告)号：EP1260586A2

公开(公告)日：2002-11-27

申请号：EP02013004.3

申请日：1995-02-23

申请人： RIBOZYME PHARMACEUTICALS, INC.

发明人： Stinchcomb, Dan T. ,  Dudycz, Lech W. ,  Chowrira, Bharat ,  Grimm, Susan ,  Direnzo, Anthony ,  Karpeisky, Alexander ,  Draper, Kenneth G. ,  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. ,  Woolf, Tod

IPC分类号： C12N15/52 ,  C12N9/00 ,  A61K31/70 ,  C07H19/04 ,  C07H19/10 ,  C07H19/20 ,  C12N15/10 ,  A61K48/00 ,  C12N15/86 ,  C12N15/87

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： Enzymatic RNA molecules which cleave ICAM-1 mRNA, IL-5 mRNA, rel A mRNA, TNF-α mRNA, RSV mRNA or RSV genomic RNA, or CML associated mRNA, and use of these molecules for the treatment of pathological conditions related to those mRNA-levels; ribonucleosides or nucleotides modified in 2', 3' or 5', methods for their synthesis, purification and deprotection; vectors containing multiple enzymatic nucleic acids, optionally in chimeric form with tRNAs; method for introducing enzymatic nucleic acids into cells by forming a complex with a second nucleic acid, where the complex is capable of taking an R-loop base-paired structure; method for altering a mutant nucleic acid in vivo by hybridization with an oligonucleotide capable of activating ds RNA deaminase, comprising an enzymatic activity or a chemical mutagen. Further are disclosed trans-cleaving or -ligating hairpin ribozymes lacking a substrate RNA moiety, as well as hammerhead ribozymes having an interconnecting loop between base pairs in stem II.

摘要翻译： 酶促RNA分子，其裂解ICAM-1的mRNA，IL-5的mRNA，REL甲mRNA的，TNF-α的mRNA RSV mRNA或基因组RSV RNA，或CML相关的mRNA，并使用合成分子为与这些治疗的病理状况 的mRNA的水平; 核糖核苷或2的修饰的核苷酸“ 3' 或5”，它们的合成方法，纯化和脱保护; 含有载体的多个酶促核酸，任选地嵌合形式与tRNA的; 方法，用于通过形成与第二核酸，其中所述复合物是能够承担R-环路碱基配对结构的复合引入酶促核酸引入细胞; 方法用于通过杂交改变体内的突变体的核酸与能够dsRNA的脱氨酶的活化的寡核苷酸，包括酶活性或化学诱变剂的。 进一步是游离缺失光盘反式裂解核酶或-ligating发夹缺乏RNA底部分，以及锤头状核酶具有在干II互连碱基对之间的循环。

公开(公告)号：EP0725788B1

公开(公告)日：1998-12-16

申请号：EP94932003.0

申请日：1994-10-24

申请人： RIBOZYME PHARMACEUTICALS, INC.

发明人： DUDYCZ, Lech, W.

IPC分类号： C07H21/00

CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C07H21/00 ,  C12N15/101 ,  C12N15/1131 ,  C12N15/1136 ,  C12N15/1138 ,  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

公开(公告)号：EP0746614A1

公开(公告)日：1996-12-11

申请号：EP95909920.0

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. ,  KODAK, Anil ,  PAVCO, Pamela ,  BEIGELMAN, Leonid ,  SULLIVAN, Sean M. ,  SWEEDLER, David ,  THOMPSON, James D. ,  TRACZ, Danuta ,  USMAN, Nassim ,  WINCOTT, Francine E. ,  WOOLF, Tod

IPC分类号： C12N15 ,  A61K38 ,  A61K48

CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C12N15/101 ,  C12N15/1131 ,  C12N15/1136 ,  C12N15/1138 ,  C12N15/85 ,  C12N15/86 ,  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： Enzymatic RNA molecules which cleave ICAM-1 mRNA, IL-5 mRNA, rel A mRNA, TNF-α mRNA, RSV mRNA or RSV genomic RNA, or CML associated mRNA, and use of these molecules for the treatment of pathological conditions related to those mRNA-levels; ribonucleosides or nucleotides modified in 2', 3' or 5', methods for their synthesis, purification and deprotection; vectors containing multiple enzymatic nucleic acids, optionally in chimeric form with tRNAs; method for introducing enzymatic nucleic acids into cells by forming a complex with a second nucleic acid, where the complex is capable of taking an R-loop base-paired structure; method for altering a mutant nucleic acid in vivo by hybridization with an oligonucleotide capable of activating dsRNA deaminase, comprising an enzymatic activity or a chemical mutagen. Further are disclosed trans-cleaving or -ligating hairpin ribozymes lacking a substrate RNA moiety, as well as hammerhead ribozymes having an interconnecting loop between base pairs in stem II.

公开(公告)号：EP0590082A4

公开(公告)日：1995-04-12

申请号：EP92914930

申请日：1992-06-11

申请人： ISIS PHARMACEUTICALS INC

发明人： MONIA BRETT P ,  FREIER SUSAN M ,  ECKER DAVID J

IPC分类号： A61K31/70 ,  A61K38/00 ,  A61K48/00 ,  C07K14/82 ,  C12N9/02 ,  C12N15/09 ,  C12N15/113 ,  C12N15/12 ,  C12Q1/68 ,  C12N15/11

CPC分类号： C12N15/1135 ,  A61K38/00 ,  C07K14/82 ,  C07K2319/00 ,  C07K2319/61 ,  C12N9/0069 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/3521 ,  C12N2310/3527 ,  C12N2310/3533 ,  C12N2310/3535 ,  C12Q1/6886 ,  C12Q2600/156

公开(公告)号：EP0604409A1

公开(公告)日：1994-07-06

申请号：EP91903066.0

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： A61K31 ,  A61K48 ,  A61P31 ,  A61P43 ,  C07H19 ,  C07H21 ,  C07H23 ,  C07J43 ,  C07K14 ,  C12N9 ,  C12N15 ,  C12P21 ,  C12Q1 ,  A61K38

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： On décrit des compositions et des procédés servant à moduler l'activité ARN et ADN. Dans les modes de réalisation préférés, on prépare des compositions non codantes comprenant des parties cibles et des parties réactives. On utilise de préférence des parties réactives qui agissent par le clivage des liaisons phosphorodiesters, par le clivage des liaisons des sucres du squelette, ou par la modification des bases. Les groupes qui améliorent les caractéristiques pharmacolodynamiques et pharmacocinétiques des oligonucléotides sont également utiles dans certains modes de réalisation. On effectue également de préférence l'apport des fonctionnalités réactive et non réactive au sillon mineur formé par l'hybridation de la composition avec l'ARN cible. On décrit également des procédés thérapeutiques, diagnostiques et de recherche. On a prévu des nucléosides thérapeutiques, diagnostiques et de recherche. On a prévu des nucléosides synthétiques et des fragments nucléosides synthétiques utiles à l'élaboration des oligonucléotides et des analogues oligonucléotidiques.

公开(公告)号：EP2841578B1

公开(公告)日：2017-06-07

申请号：EP13720617.3

申请日：2013-04-23

申请人： BioMarin Technologies B.V.

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

IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P25/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/346 ,  C12N2310/3521 ,  C12N2320/34

摘要： The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.

摘要翻译： 当前发明提供了改进的寡核苷酸和其用于治疗，改善，预防，延缓和/或治疗相关的遗传神经肌肉或神经变性病症的人顺式元件重复不稳定性的用途。

公开(公告)号：EP3098314A1

公开(公告)日：2016-11-30

申请号：EP16179166.0

申请日：2012-08-30

申请人： Arrowhead Research Corporation

发明人： CHEN, Jinyun ,  GAMPA, Kalyani ,  HUESKEN, Dieter ,  STEGMEIER, Frank P. ,  STUMP, Mark ,  VARGEESE, Chandra ,  WIELER, Jan ,  ZHOU, Wenlai

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P35/00 ,  A61P31/12 ,  A61P37/06

CPC分类号： C12N15/113 ,  A61K31/5377 ,  A61K31/713 ,  A61K45/06 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/351 ,  C12N2320/31 ,  C12N2310/3521 ,  C12N2310/3525

摘要： The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer, autoimmune and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.

摘要翻译： 本公开涉及使用治疗有效量的RNAi剂对HSF治疗热休克因子1（HSF1）相关疾病如癌症，自身免疫性和病毒性疾病的方法。

公开(公告)号：EP2739637A4

公开(公告)日：2015-04-22

申请号：EP12819429

申请日：2012-08-03

申请人： QUARK PHARMACEUTICALS INC

发明人： FEINSTEIN ELENA ,  AVKIN-NACHUM SHARON ,  METT IGOR ,  KALINSKI HAGAR

IPC分类号： C07H21/00 ,  A61K31/7088 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/332 ,  C12N2310/333 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/337 ,  C12N2310/3521 ,  C12N2310/50

公开(公告)号：EP2841578A1

公开(公告)日：2015-03-04

申请号：EP13720617.3

申请日：2013-04-23

申请人： Prosensa Technologies B.V.

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

IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P25/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/346 ,  C12N2310/3521 ,  C12N2320/34

摘要： The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.