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1. 发明公开

EP1383782A1 OLIGONUCLEOTIDE MEDIATED INHIBITION OF HEPATITIS B VIRUS AND HEPATITIS C VIRUS REPLICATION 审中-公开
标题翻译：寡核苷酸抑制丙型复制 - B-病毒和丙型肝炎病毒
公开(公告)号：EP1383782A1
公开(公告)日：2004-01-28
申请号：EP02728566.7
申请日：2002-03-26
申请人： SIRNA THERPEUTICS, INC. , Blatt, Lawrence , Macejak, Dennis , McSwiggen, James , Morrissey, David , Pavco, Pamela
发明人： BLATT, Lawrence , MACEJAK, Dennis , MCSWIGGEN, James , MORRISSEY, David , PAVCO, Pamela , LEE, Patrice , DRAPER, Kenneth , ROBERTS, Elisabeth
IPC分类号： C07H21/02 , C07H21/04 , A01K67/00 , A01K67/033 , A01K67/027 , G01N33/00
CPC分类号： C12N15/111 , A61K38/21 , C07H21/02 , C07H21/04 , C12N15/1131 , C12N2310/11 , C12N2310/12 , C12N2310/13 , C12N2310/16 , C12N2310/315 , C12N2310/317 , C12N2310/319 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/3519 , C12N2320/31 , C12N2320/51 , C12Q1/706 , A61K2300/00 , C12N2310/3521
摘要： The present invention relates to nucleic acid molecules, including antisense and enzymatic nucleic acid molecules, such as hammerhead ribozymes, DNAzymes, Inozymes, Zinzymes, Amberzymes, and G-cleaver ribozymes, which modulate the synthesis, expression and/or stability of an HCV or HBV RNA and methods for their use alone or in combination with other therapies. In addition, nucleic acid decoy molecules and aptamers that bind to HBV reverse transcriptase and/or HBV reverse transcriptase primer sequences and methods for their use alone or in combination with other therapies, are disclosed. Oligonucleotides that specifically bind the Enhancer I region of HBV DNA are further disclosed. The present invention further relates to the use of nucleic acids, such as decoy and aptamer molecules of the invention, to modulate the expression of Hepatitis B virus (HBV) genes and HBV viral replication. Furthermore, HBV animal models and methods of use are disclosed, including methods of screening for compounds and/or potential therapies directed against HBV. The present invention also relates to compounds, including enzymatic nucleic acid molecules, ribozymes, DNAzymes, nuclease activating compounds and chimeras such as 2',5'-adenylates, that modulate the expression and/or replication of hepatitis C virus (HCV).

2. 发明专利

AU2004100777A4 Tool for Moving a 4WD Manual Locking Hub 未知
公开(公告)号：AU2004100777A4
公开(公告)日：2004-11-04
申请号：AU2004100777
申请日：2004-09-16
申请人： MORRISSEY DAVID JAMES
发明人： MORRISSEY DAVID JAMES
IPC分类号： B25B13/48 , B60K23/08

3. 发明申请

WO2003070750A3 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS 审中-公开
公开(公告)号：WO2003070750A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/005043
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS, INC , McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , MORRISSEY, David
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , MORRISSEY, David
IPC分类号： C12Q1/68
摘要： The present invention concerns methods and reagents useful in modulating hepatitis C virus (HCV) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 capable of mediating RNA interference (RNA/) against hepatitis C virus (HCV) gene expression and/or activity. The small nucleic acid molecules are useful in the treatment and diagnosis of HCV infection, liver failure, hepatocellular carcinoma, cirrhosis and any other disease or condition that responds to modulation of HCV expression or activity.

4. 发明申请

WO2007076328A3 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS 审中-公开
标题翻译： RNA干扰介导的HEPATITIS C病毒的抑制
公开(公告)号：WO2007076328A3
公开(公告)日：2008-08-14
申请号：PCT/US2006062252
申请日：2006-12-18
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT
发明人： MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT
IPC分类号： A61K9/127 , A61K31/713 , C12N15/11 , C12N15/113
CPC分类号： C12N15/1131 , A61K31/713 , A61K47/6911 , C12N15/111 , C12N2310/14 , C12N2310/318 , C12N2310/51 , C12N2770/24211
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including 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 capable of mediating RNA interference (RNAi) against gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticlc (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对基因表达和/或活性调节作出反应的性状，疾病和病症的化合物，组合物和方法。本发明还涉及与涉及参与基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症相关的化合物，组合物和方法，或调节其维持或发展的其他细胞过程性状，疾病和状况。具体地说，本发明涉及包括小核酸分子如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和双链RNA 能够介导针对基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸分子的这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源性微RNA（miRNA）（例如miRNA抑制剂）或内源性短干扰RNA（例如miRNA抑制剂）的内源性RNA分子的功能的小核酸分子，例如siNA，siRNA等， siRNA）（例如siRNA抑制剂）或可以通过干扰与这些内源性RNA（例如RISC）相关的内源性RNA或蛋白质的调节功能来调节基因表达来调节基因表达（例如，RISC抑制剂），包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子并且可用于例如提供组合物以预防，抑制或减少与受试者或生物体中的基因表达或活性相关的疾病，性状和病症。

5. 发明申请

WO2003070918A3 RNA INTERFERENCE BY MODIFIED SHORT INTERFERING NUCLEIC ACID 审中-公开
公开(公告)号：WO2003070918A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/005346
申请日：2003-02-20
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
IPC分类号： C07H21/04
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified 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 capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

6. 发明申请

WO2003070918A2 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID 审中-公开
标题翻译： RNA干扰介导的基因表达的抑制使用化学修饰的短暂干扰核酸
公开(公告)号：WO2003070918A2
公开(公告)日：2003-08-28
申请号：PCT/US2003/005346
申请日：2003-02-20
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
IPC分类号： C12N
CPC分类号： C12N15/8218 , A61K38/00 , A61K47/54 , C07H21/02 , C12N15/111 , C12N15/1131 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/51 , C12N2330/30 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified 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 capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
摘要翻译：本发明涉及可用于调节各种应用中的基因表达的方法和试剂，包括在治疗，诊断，靶标验证和基因组发现应用中的用途。具体地，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。小核酸分子可用于治疗对细胞，组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。

7. 发明申请

WO2011145081A1 INFLUENZA VIRUS REASSORTMENT METHOD 审中-公开
标题翻译：流感病毒重新排毒方法
公开(公告)号：WO2011145081A1
公开(公告)日：2011-11-24
申请号：PCT/IB2011/052218
申请日：2011-05-20
申请人： NOVARTIS AG , TRUSHEIM, Heidi , MASON, Peter , FRANTI, Michael , KEINER, Bjoern , SACKAL, Melissa , HUNZIKER, Juerg , MORRISSEY, David , NATT, François Jean-Charles
发明人： TRUSHEIM, Heidi , MASON, Peter , FRANTI, Michael , KEINER, Bjoern , SACKAL, Melissa , HUNZIKER, Juerg , MORRISSEY, David , NATT, François Jean-Charles
IPC分类号： A61K39/145 , C12N15/113 , C12N7/02
CPC分类号： C12N7/00 , A61K39/12 , A61K39/145 , A61K2039/525 , C12N15/1131 , C12N2310/14 , C12N2320/00 , C12N2760/16134 , C12N2760/16151 , C12N2760/16152
摘要： Methods for producing reassortant viruses are provided wherein the transcription and/or translation of the hemagglutinin and/or neuraminidase genes are suppressed.
摘要翻译：提供了产生重配病毒的方法，其中抑制了血凝素和/或神经氨酸酶基因的转录和/或翻译。

8. 发明申请

WO2008103276A3 COMPOSITIONS AND METHODS FOR POTENTIATED ACTIVITY OF BIOLOGICALY ACTIVE MOLECULES 审中-公开
公开(公告)号：WO2008103276A3
公开(公告)日：2008-08-28
申请号：PCT/US2008/002006
申请日：2008-02-15
申请人： MERCK & CO., INC. , JADHAV, Vasant , VARGEESE, Chandra , SHAW, Lucinda , MORRISSEY, David , JENSEN, Kristi
发明人： JADHAV, Vasant , VARGEESE, Chandra , SHAW, Lucinda , MORRISSEY, David , JENSEN, Kristi
IPC分类号： A61K31/70
摘要： The present invention relates to novel compositions and methods for potentiating the activity of biologically active molecules in conjunction with one or more delivery vehicles and one or more carrier molecules. Specifically, the invention features the use of a carrier molecule in combination with a delivery vehicle and a biologically active molecule of interest to potentiate the activity of the biologically active molecule. The carrier molecule can be biologically inert, inactive, or attenuated; or can alternately be biologically active in the same or different manner than the biologically active molecule of interest. Specifically, the invention features novel particle forming delivery agents including cationic lipids, microparticles, and nanoparticles that are useful for delivering various biologically active molecules to cells in conjunction with a carrier molecule. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism that are delivered intracellularly in conjunction with a carrier molecule. In various embodiments, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, allozymes, aptamers, decoys and analogs thereof, and 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, to relevant cells and/or tissues, such as in a subject or organism, in conjunction with one or more carrier molecules. Such novel cationic lipids, microparticles, nanoparticles and transfection agents that are used in conjuction with one or more carrier molecules are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism.

9. 发明申请

WO2007076328A2 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制HEPATITIS C病毒（HCV）基因表达使用短暂的干扰核酸（siNA）
公开(公告)号：WO2007076328A2
公开(公告)日：2007-07-05
申请号：PCT/US2006/062252
申请日：2006-12-18
申请人： SIRNA THERAPEUTICS, INC. , McSWIGGEN, James , MORRISSEY, David , GUERCIOLINI, Roberto , VARGEESE, Chandra , JADHAV, Vasant
发明人： McSWIGGEN, James , MORRISSEY, David , GUERCIOLINI, Roberto , VARGEESE, Chandra , JADHAV, Vasant
IPC分类号： C12N15/11 , A61K31/713 , A61K9/127
CPC分类号： C12N15/1131 , A61K31/713 , A61K47/6911 , C12N15/111 , C12N2310/14 , C12N2310/318 , C12N2310/51 , C12N2770/24211
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including 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 capable of mediating RNA interference (RNAi) against gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticlc (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对基因表达和/或活性调节作出反应的性状，疾病和病症的化合物，组合物和方法。本发明还涉及与涉及参与基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症相关的化合物，组合物和方法，或调节其维持或发展的其他细胞过程性状，疾病和状况。具体地说，本发明涉及包括小核酸分子如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和双链RNA 能够介导针对基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸分子的这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源性微RNA（miRNA）（例如miRNA抑制剂）或内源性短干扰RNA（例如miRNA抑制剂）的内源性RNA分子的功能的小核酸分子，例如siNA，siRNA等， siRNA）（例如siRNA抑制剂）或可以通过干扰与这些内源性RNA（例如RISC）相关的内源性RNA或蛋白质的调节功能来调节基因表达来调节基因表达（例如，RISC抑制剂），包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子并且可用于例如提供组合物以预防，抑制或减少与受试者或生物体中的基因表达或活性相关的疾病，性状和病症。

10. 发明申请

WO2005019453A3 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的化学修饰短介导核酸（siNA）抑制基因表达
公开(公告)号：WO2005019453A3
公开(公告)日：2005-06-23
申请号：PCT/US2004016390
申请日：2004-05-24
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , MORRISSEY DAVID , ZINNEN SHAWN , JADHAV VASANT , VAISH NARENDRA
发明人： MCSWIGGEN JAMES , MORRISSEY DAVID , ZINNEN SHAWN , JADHAV VASANT , VAISH NARENDRA
IPC分类号： A01N43/04 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12P19/34 , C12N15/11
CPC分类号： C12N15/113 , C12N2310/14
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, cosmetic, cosmeceutical, prophylactic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified 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 capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease (e.g., cancer, proliferative, inflammatory, metabolic, autoimmune, nuerologic, ocular diseases), condition, trait (e.g., hair growth and removal), genotype or phenotype that responds to modulation of gene expression or activity in a cell, tissue, or organism. Such small nucleic acid molecules can be administered systemically, locally, or topically.
摘要翻译：本发明涉及用于调节各种应用中的基因表达的方法和试剂，包括用于治疗，美容，药妆，预防，诊断，靶标验证和基因组发现应用。具体而言，本发明涉及合成的化学修饰的小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA （shRNA）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。小核酸分子可用于治疗任何疾病（例如癌症，增殖性，炎性，代谢性，自身免疫性，神经性，眼部疾病），病症，性状（例如毛发生长和去除），基因型或表型，调节细胞，组织或生物体中的基因表达或活性。这样的小核酸分子可以全身，局部或局部施用。

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