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    RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    21.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 有权

    公开(公告)号：US20090306184A1

    公开(公告)日：2009-12-10

    申请号：US12418477

    申请日：2009-04-03

    申请人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini ,  Chandra Vargeese ,  Vasant Jadhav

    发明人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini ,  Chandra Vargeese ,  Vasant Jadhav

    IPC分类号： A61K31/7105 ,  C07H21/02

    CPC分类号： C12N15/1131 ,  A61K47/6911 ,  C12N2310/14 ,  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 nanoparticle (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.

    RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
    22.
    发明授权
    RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) 有权
    使用短干扰核酸（siNA）的RNA干扰介导的丙型肝炎病毒（HCV）表达的抑制

    公开(公告)号：US07935812B2

    公开(公告)日：2011-05-03

    申请号：US12418477

    申请日：2009-04-03

    申请人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini ,  Chandra Vargeese ,  Vasant Jadhav

    发明人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini ,  Chandra Vargeese ,  Vasant Jadhav

    IPC分类号： C07H21/04 ,  C07H21/02 ,  A61K48/00

    CPC分类号： C12N15/1131 ,  A61K47/6911 ,  C12N2310/14 ,  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 hepatitis C virus (HCV) 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 hepatitis C virus (HCV) 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 capable of mediating or that mediate RNA interference (RNAi) against hepatitis C virus (HCV) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.

    摘要翻译： 本发明涉及用于研究，诊断和治疗对丙型肝炎病毒（HCV）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。 本发明还涉及与涉及丙型肝炎病毒（HCV）基因表达途径或介导的其他细胞过程的基因的表达和/或活性的调节作用的性状，疾病和病症有关的化合物，组合物和方法 维持或发展这些性状，疾病和状况。 具体地，本发明涉及能够介导或介导针对丙型肝炎病毒（HCV）基因表达的RNA干扰（RNAi）的双链核酸分子，包括这种小核酸分子的混合物和这种小的核酸分子的脂质纳米颗粒（LNP）制剂 核酸分子。

    RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
    23.
    发明申请
    RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA) 审中-公开

    公开(公告)号：US20080207542A1

    公开(公告)日：2008-08-28

    申请号：US11510872

    申请日：2006-08-25

    申请人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini ,  Chandra Vargeese ,  Vasant Jadhav

    发明人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini ,  Chandra Vargeese ,  Vasant Jadhav

    IPC分类号： A61K31/713

    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 nanoparticle (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.

    CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE
    24.
    发明申请
    CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE 审中-公开

    公开(公告)号：US20090176725A1

    公开(公告)日：2009-07-09

    申请号：US12064014

    申请日：2006-08-17

    申请人： David Morrissey ,  James McSwiggen ,  Leonid Beigelman

    发明人： David Morrissey ,  James McSwiggen ,  Leonid Beigelman

    IPC分类号： A61K31/713 ,  C07H21/04

    CPC分类号： A61K31/70 ,  A61K48/00 ,  C07H21/02

    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 nanoparticle (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.

    COMPOSITIONS AND METHODS FOR POTENTIATED ACTIVITY OF BIOLOGICALLY ACTIVE MOLECULES
    27.
    发明申请
    COMPOSITIONS AND METHODS FOR POTENTIATED ACTIVITY OF BIOLOGICALLY ACTIVE MOLECULES 审中-公开

    公开(公告)号：US20100015218A1

    公开(公告)日：2010-01-21

    申请号：US12526869

    申请日：2008-02-15

    申请人： Vasant Jadhav ,  Chandra Vargeese ,  Lucinda Shaw ,  David Morrissey ,  Kristi Jensen

    发明人： Vasant Jadhav ,  Chandra Vargeese ,  Lucinda Shaw ,  David Morrissey ,  Kristi Jensen

    IPC分类号： A61K9/127 ,  A61K31/7052 ,  C12N15/63 ,  A61P43/00

    CPC分类号： C12N15/88

    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 conjunction 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.

    RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
    28.
    发明授权
    RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA) 失效
    RNA干扰介导的使用短干扰核酸（siNA）抑制血管内皮生长因子和血管内皮生长因子受体基因表达

    公开(公告)号：US07517864B2

    公开(公告)日：2009-04-14

    申请号：US11299391

    申请日：2005-12-09

    申请人： Chandra Vargeese ,  Vasant Jadhav ,  David Morrissey

    发明人： Chandra Vargeese ,  Vasant Jadhav ,  David Morrissey

    IPC分类号： A61K48/00 ,  C07H21/02 ,  C07H21/04

    CPC分类号： C12N15/1138 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/332 ,  C12N2320/32

    摘要： This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR gene 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 VEGF and/or VEGFR 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 VEGF and/or VEGFR genes. The application also relates to methods of treating diseases and conditions associated with VEGF and/or VEGFR gene expression, such as ocular diseases and conditions, including age related macular degeneration (AMD) and diabetic retinopathy, as well as providing dosing regimens and treatment protocols.

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节VEGF和/或VEGFR基因表达的化合物，组合物和方法。 本发明还涉及使用小核酸分子调节参与VEGF和/或VEGFR基因表达和/或活性通过RNA干扰（RNAi）的其它基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，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） ）分子和用于调节VEGF和/或VEGFR基因表达的方法。 该应用还涉及治疗与VEGF和/或VEGFR基因表达相关的疾病和病症的方法，例如眼部疾病和病症，包括年龄相关性黄斑变性（AMD）和糖尿病性视网膜病变，以及提供给药方案和治疗方案。

    Chemically modified double stranded nucleic acid molecules that mediate RNA interference
    29.
    发明申请
    Chemically modified double stranded nucleic acid molecules that mediate RNA interference 审中-公开
    介导RNA干扰的化学修饰双链核酸分子

    公开(公告)号：US20060217331A1

    公开(公告)日：2006-09-28

    申请号：US11299254

    申请日：2005-12-08

    申请人： Chandra Vargeese ,  Vasant Jadhav ,  David Morrissey

    发明人： Chandra Vargeese ,  Vasant Jadhav ,  David Morrissey

    IPC分类号： A61K48/00 ,  C07H21/02

    CPC分类号： C12N15/1137 ,  A61K31/713 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/14 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/53 ,  C12N2310/3521

    摘要： This invention relates to compounds, compositions, and methods useful for modulating gene expression using chemically modified double stranded nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of genes via RNA interference (RNAi). The invention features various modifications to double stranded nucleic acid structures, including chemically modified overhangs and optimized stabilization motifs of guide (antisense) strand and passenger (sense) strands of double stranded nucleic acid molecules that allow for potent RNA interference in therapeutically relevant applications.

    摘要翻译： 本发明涉及使用化学修饰的双链核酸分子调节基因表达的化合物，组合物和方法。 特别地，本发明的特征在于包括小核酸分子的双链核酸分子，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）分子和用于通过RNA干扰（RNAi）调节基因表达的方法。 本发明具有对双链核酸结构的各种修饰，其包括引导（反义）链的引导（反义）链和乘客（有义）链的经化学修饰的突出端和优化的稳定基序，其允许在治疗相关应用中有效的RNA干扰。