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    COMPOSITIONS AND METHODS FOR POTENTIATED ACTIVITY OF BIOLOGICALLY ACTIVE MOLECULES
    1.
    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 HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    2.
    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.

    CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE
    5.
    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.