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52 条记录 (耗时 0.065 秒)

    NOVEL TRIALKYL CATIONIC LIPIDS AND METHODS OF USE THEREOF
    41.
    发明申请
    NOVEL TRIALKYL CATIONIC LIPIDS AND METHODS OF USE THEREOF 审中-公开
    新型临床阳离子脂肪及其使用方法

    公开(公告)号：US20140288146A1

    公开(公告)日：2014-09-25

    申请号：US13870951

    申请日：2013-04-25

    申请人： PROTIVA BIOTHERAPEUTICS, INC.

    发明人： James Heyes ,  Mark Wood ,  Alan Martin

    IPC分类号： A61K47/44 ,  C07C271/20 ,  A61K31/7088 ,  C07C229/12

    CPC分类号： A61K47/44 ,  A61K9/1272 ,  A61K31/7088 ,  A61K31/7105 ,  A61K31/713 ,  A61K47/34 ,  C07C229/12 ,  C07C271/20

    摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.

    摘要翻译： 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地，这些包括提供核酸的有效包封的新型阳离子脂质和核酸 - 脂质颗粒，并将包封的核酸在体内有效递送至细胞。 本发明的组合物是高度有效的，从而允许以相对低的剂量有效击倒特异性靶蛋白。 此外，与本领域已知的组合物和方法相比，本发明的组合物和方法毒性低，并提供更大的治疗指数。

    SILENCING OF POLO-LIKE KINASE EXPRESSION USING INTERFERING RNA
    47.
    发明申请
    SILENCING OF POLO-LIKE KINASE EXPRESSION USING INTERFERING RNA 审中-公开

    公开(公告)号：US20160115483A1

    公开(公告)日：2016-04-28

    申请号：US14638885

    申请日：2015-03-04

    申请人： PROTIVA BIOTHERAPEUTICS, INC.

    发明人： IAN MACLACHLAN ,  ADAM JUDGE

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1137 ,  A61K9/0019 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  C12N15/88 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/335 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/30 ,  C12Y207/11021

    摘要： The present invention provides compositions comprising interfering RNA (e.g., siRNA, aiRNA, miRNA) that target polo-like kinase 1 (PLK-1) expression and methods of using such compositions to silence PLK-1 expression. More particularly, the present invention provides unmodified and chemically modified interfering RNA molecules which silence PLK-1 expression and methods of use thereof. The present invention also provides serum-stable nucleic acid-lipid particles (e.g., SNALP) comprising an interfering RNA molecule described herein, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The present invention further provides methods of silencing PLK-1 gene expression by administering an interfering RNA molecule described herein to a mammalian subject. The present invention additionally provides methods of identifying and/or modifying PLK-1 interfering RNA having immunostimulatory properties. Methods for sensitizing a cell such as a cancer cell to the effects of a chemotherapy drug comprising sequentially delivering PLK-1 interfering RNA followed by the chemotherapy drug are also provided.

    Compositions and methods for silencing SMAD4
    49.
    发明授权
    Compositions and methods for silencing SMAD4 有权
    SMAD4沉默的组成和方法

    公开(公告)号：US09035039B2

    公开(公告)日：2015-05-19

    申请号：US13725796

    申请日：2012-12-21

    申请人： Protiva Biotherapeutics Inc.

    发明人： Ammen P. Dhillon ,  Amy C. H. Lee ,  Ian MacLachlan

    IPC分类号： C07H21/04 ,  C07H21/02 ,  A61K31/7105 ,  A61K31/713

    CPC分类号： C07H21/02 ,  A61K31/7105 ,  A61K31/713

    摘要： The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA (e.g., dsRNA such as siRNA) that target SMAD4 gene expression, lipid particles comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles (e.g., for treating anemia of inflammation in humans).

    摘要翻译： 本发明提供了包含靶向SMAD4基因表达的治疗性核酸例如干扰RNA（例如dsRNA例如siRNA）的组合物，包含治疗性核酸的一种或多种（例如混合物）的脂质颗粒，制备该脂质的方法 颗粒和递送和/或施用脂质颗粒的方法（例如，用于治疗人类的炎症性贫血）。

    COMPOSITIONS AND METHODS FOR SILENCING ALDEHYDE DEHYDROGENASE
    50.
    发明申请
    COMPOSITIONS AND METHODS FOR SILENCING ALDEHYDE DEHYDROGENASE 有权
    用于沉淀醛脱氢酶的组合物和方法

    公开(公告)号：US20140248338A1

    公开(公告)日：2014-09-04

    申请号：US14349619

    申请日：2012-10-04

    申请人： Protiva Biotherapeutics, Inc.

    发明人： Ian MacLachlan ,  Amy C.H. Lee

    IPC分类号： C12N15/113 ,  A61K9/127 ,  A61K31/713

    CPC分类号： A61K9/127 ,  A61K31/713 ,  C07H21/02 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/3521 ,  C12Y102/01003

    摘要： The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA (e.g., dsRNA such as siRNA) that target aldehyde dehydrogenase (ALDH) gene expression, lipid particles comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles (e.g., for treating alcoholism in humans).

    摘要翻译： 本发明提供包含靶向醛脱氢酶（ALDH）基因表达的治疗性核酸例如干扰RNA（例如，dsRNA例如siRNA）的组合物，包含治疗性核酸的一种或多种（例如混合物）的脂质颗粒，方法 制备脂质颗粒，以及递送和/或施用脂质颗粒（例如，用于治疗人类酒精中毒）的方法。