利索-全球专利检索

  1. 登录
  2. 注册

搜索结果

53 条记录 (耗时 0.017 秒)

    METHODS AND COMPOSITIONS FOR NUCLEIC ACID AND PROTEIN PAYLOAD DELIVERY
    4.
    发明申请
    METHODS AND COMPOSITIONS FOR NUCLEIC ACID AND PROTEIN PAYLOAD DELIVERY 审中-公开

    公开(公告)号：WO2018112278A1

    公开(公告)日：2018-06-21

    申请号：PCT/US2017/066541

    申请日：2017-12-14

    申请人： LIGANDAL, INC.

    发明人： WATSON, Andre Ronald ,  FOSTER, Christian

    IPC分类号： A61K47/00 ,  A61K47/30 ,  A61K47/50 ,  A61K47/51

    摘要： Provided are methods and compositions for delivering a nucleic acid, protein, and/or ribonucleoprotein payload to a cell. Also provided are delivery molecules that include a peptide targeting ligand conjugated to a protein or nucleic acid payload (e.g., an siRNA molecule), or conjugated to a charged polymer polypeptide domain (e.g., poly-arginine such as 9R or a poly-histidine such as 6H, and the like). The targeting ligand provides for (i) targeted binding to a cell surface protein, and (ii) engagement of a long endosomal recycling pathway. As such, when the targeting ligand engages the intended cell surface protein, the delivery molecule enters the cell (e.g., via endocytosis) but is preferentially directed away from the lysosomal degradation pathway.

    METHOD TO ANALYZE AND OPTIMIZE GENE EDITING MODULES AND DELIVERY APPROACHES
    5.
    发明申请
    METHOD TO ANALYZE AND OPTIMIZE GENE EDITING MODULES AND DELIVERY APPROACHES 审中-公开
    方法分析和优化基因编辑模块和交付方法

    公开(公告)号：WO2018060238A1

    公开(公告)日：2018-04-05

    申请号：PCT/EP2017/074480

    申请日：2017-09-27

    申请人： F. HOFFMANN-LA ROCHE AG ,  HOFFMANN-LA ROCHE INC.

    发明人： BRINKMANN, Ulrich ,  KILLIAN, Tobias

    IPC分类号： C12N15/10 ,  A61K47/51

    摘要： Herein is reported a method for determining the introduction of a nucleic acid into the genome of a mammalian cell, whereby the mammalian cell comprises one or two transcriptionally active alleles of a DPH1, DPH2, DPH4 and/or DPH5 gene, comprising the steps of transfecting the mammalian cell with one or more plasmids comprising the nucleic acid to be introduced, and the elements required for gene editing of said DPH gene, cultivating the transfected cell in the presence of a DPH gene transcription sensitive toxin, and thereby determining the introduction of a nucleic acid into the genome of the mammalian cell if the transfected cells is viable in the presence of the toxin.

    摘要翻译： 本文报道了用于确定将核酸引入哺乳动物细胞基因组中的方法，其中哺乳动物细胞包含DPH1，DPH2，DPH4的一种或两种转录活性等位基因和/或 DPH5基因的方法，包括用一种或多种包含待导入核酸的质粒和所述DPH基因的基因编辑所需元件转染哺乳动物细胞的步骤，在DPH基因转录敏感毒素存在下培养转染细胞 ，从而确定如果转染的细胞在毒素存在下存活，则将核酸导入哺乳动物细胞的基因组中。

    DRUG DELIVERY USING APTAMER CONSTRUCT
    6.
    发明申请
    DRUG DELIVERY USING APTAMER CONSTRUCT 审中-公开
    使用APTAMER构造的药物递送

    公开(公告)号：WO2018055360A1

    公开(公告)日：2018-03-29

    申请号：PCT/GB2017/052795

    申请日：2017-09-20

    申请人： IMPERIAL INNOVATIONS LIMITED

    发明人： ALMQUIST, Benjamin David ,  STEJSKALOVA, Anna

    IPC分类号： A61K47/34 ,  A61K47/51 ,  A61K47/42 ,  A61P17/02 ,  A61P35/00

    摘要： A molecular complex comprising a therapeutic agent and a controlled release construct, the controlled release construct comprising a primary matrix conjugation site which is linked to an cell adhesive site, via a binding region and optionally via one or more spacer elements, wherein the binding region has a folded configuration in which it is bound to the therapeutic agent, wherein the construct is configured such that when mechanical tension is applied between the primary matrix conjugation site and the cell adhesive site, the binding region adopts a less folded configuration in which bound therapeutic agent is released. Related controlled release constructs for loading with the therapeutic agent, pharmaceutical compositions and methods of manufacture and use.

    摘要翻译： 包含治疗剂和控释组合物的分子复合物，所述控释组合物包含与细胞粘附位点连接的初级基质缀合位点，经由结合区并任选地经由一个或多个 间隔元件，其中所述结合区具有折叠构型，在所述折叠构型中，所述结合区与治疗剂结合，其中所述构造构造成使得当在所述主基质缀合部位和所述细胞粘附部位之间施加机械张力时，所述结合区域采用 其中结合的治疗剂被释放的折叠较少的构型。 用于加载治疗剂的相关受控释放构建体，药物组合物以及制造和使用方法。