公开(公告)号：EP3100718B1

公开(公告)日：2019-11-27

申请号：EP16163003.3

申请日：2008-12-31

申请人： Arbutus Biopharma Corporation ,  The University of British Columbia ,  Alnylam Pharmaceuticals, Inc.

发明人： HOPE, Michael, J. ,  SEMPLE, Sean C. ,  CHEN, Jianxin ,  MADDEN, Thomas D. ,  MUI, Barbara ,  CULLIS, Pieter R. ,  CIUFOLINI, Marco A. ,  WONG, Kim F. ,  MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil G. ,  SRINIVASULU, Masunu

IPC分类号： A61K9/127 ,  A61K31/7088 ,  C07D317/28 ,  C07C229/30

公开(公告)号：EP3100718A1

公开(公告)日：2016-12-07

申请号：EP16163003.3

申请日：2008-12-31

申请人： Arbutus Biopharma Corporation ,  The University of British Columbia ,  Alnylam Pharmaceuticals

发明人： HOPE, Michael, J. ,  SEMPLE, Sean C. ,  CHEN, Jianxin ,  MADDEN, Thomas D. ,  MUI, Barbara ,  CULLIS, Pieter R. ,  CIUFOLINI, Marco A. ,  WONG, Kim F. ,  MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil G. ,  SRINIVASULU, Masunu

IPC分类号： A61K9/127 ,  A61K31/7088 ,  C07D317/28 ,  C07C229/30

摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel 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 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： 本发明提供了用于将治疗剂递送至细胞的组合物和方法。 具体而言，这些包括新型脂质和核酸 - 脂质颗粒，其提供有效的核酸包封和将包封的核酸有效递送至体内细胞。 本发明的组合物非常有效，从而允许以相对低的剂量有效敲减特定靶蛋白。 另外，本发明的组合物和方法毒性较低，并且与本领域先前已知的组合物和方法相比提供了更大的治疗指数。