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    LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE MOLECULES
    3.
    发明公开
    LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE MOLECULES 审中-公开
    ON脂质纳米粒基组合物和用于将生物活性分子的方法

    公开(公告)号：EP2104740A2

    公开(公告)日：2009-09-30

    申请号：EP07873552.9

    申请日：2007-10-17

    申请人： Sirna Therapeutics, Inc.

    发明人： CHEN, Tongqian ,  VARGEESE, Chandra ,  VAGLE, Kurt ,  WANG, Weimin ,  ZHANG, Ye

    IPC分类号： C12N15/88 ,  A61K47/48 ,  A61K9/127

    CPC分类号： C12N15/88 ,  A61K9/1271 ,  A61K47/6911 ,  C07C217/28 ,  Y10S977/797

    摘要： The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. 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. Specifically, 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, dsRNA, 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), short hairpin RNA (shRNA), and RNAi inhibitor molecules, to relevant cells and/or tissues, such as in a subject or organism. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).

    CATIONIC LIPIDS AND FORMULATED MOLECULAR COMPOSITIONS CONTAINING THEM
    5.
    发明公开
    CATIONIC LIPIDS AND FORMULATED MOLECULAR COMPOSITIONS CONTAINING THEM 审中-公开
    阳离子脂质和含制定分子组成

    公开(公告)号：EP1922300A2

    公开(公告)日：2008-05-21

    申请号：EP06849713.0

    申请日：2006-02-14

    申请人： Sirna Therapeutics Inc.

    发明人： CHEN, Tongqian ,  VARGEESE, Chandra ,  VAGLE, Kurt ,  WANG, Weimin ,  ZHANG, Ye

    IPC分类号： C07C217/28 ,  C07C217/84 ,  C07J41/00

    CPC分类号： C07C217/28 ,  A61K9/0073 ,  A61K9/1271 ,  A61K9/1272 ,  A61K47/543 ,  A61K47/6911 ,  C07C217/42 ,  C07C217/58 ,  C07C239/20 ,  C07J41/00 ,  C12N15/88

    摘要： The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. 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. Specifically, 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, dsRNA, 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. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).