公开(公告)号：US20170151333A1

公开(公告)日：2017-06-01

申请号：US15261570

申请日：2016-09-09

申请人： PROTIVA BIOTHERAPEUTICS, INC.

发明人： James Heyes ,  Alan Martin ,  Mark Wood

IPC分类号： A61K47/18 ,  A61K47/22 ,  C07C229/12 ,  C07D249/04 ,  C07C217/08 ,  C07C237/06 ,  C07C327/06 ,  C07D233/60 ,  A61K31/713 ,  C07C271/20

CPC分类号： A61K47/18 ,  A61K31/713 ,  A61K47/14 ,  A61K47/183 ,  A61K47/186 ,  A61K47/22 ,  A61K48/0033 ,  C07C217/08 ,  C07C217/46 ,  C07C229/12 ,  C07C229/30 ,  C07C237/06 ,  C07C271/20 ,  C07C327/06 ,  C07C327/22 ,  C07D233/60 ,  C07D249/04 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32

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.

公开(公告)号：US20170007702A1

公开(公告)日：2017-01-12

申请号：US15150016

申请日：2016-05-09

申请人： PROTIVA BIOTHERAPEUTICS, INC.

发明人： James Heyes ,  Mark Wood ,  Alan Martin

IPC分类号： A61K47/18 ,  A61K9/14 ,  A61K31/7088 ,  A61K38/00 ,  C12N15/113

摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel, trialkyl, 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.

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三烷基，阳离子脂质和核酸 - 脂质颗粒，并且将体内核酸包被的核酸有效地递送到体内。 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。

公开(公告)号：US09352042B2

公开(公告)日：2016-05-31

申请号：US14380536

申请日：2013-02-22

申请人： PROTIVA BIOTHERAPEUTICS, INC.

发明人： James Heyes ,  Mark Wood ,  Alan Martin

IPC分类号： A61K47/18 ,  A61K9/14 ,  A61K31/713 ,  C07C229/12 ,  C07C217/08 ,  C07C271/20 ,  C12N15/113

CPC分类号： A61K47/18 ,  A61K9/145 ,  A61K31/7088 ,  A61K31/713 ,  A61K38/00 ,  C07C217/08 ,  C07C229/12 ,  C07C271/20 ,  C07C2601/02 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32

摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel, trialkyl, 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.

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三烷基，阳离子脂质和核酸 - 脂质颗粒，并且将体内核酸包被的核酸有效地递送到体内。 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。

公开(公告)号：US20150064242A1

公开(公告)日：2015-03-05

申请号：US14380536

申请日：2013-02-22

申请人： PROTIVA BIOTHERAPEUTICS, INC.

发明人： James Heyes ,  Mark Wood ,  Alan Martin

IPC分类号： A61K47/18 ,  C12N15/113 ,  A61K9/14 ,  A61K31/713

CPC分类号： A61K47/18 ,  A61K9/145 ,  A61K31/7088 ,  A61K31/713 ,  A61K38/00 ,  C07C217/08 ,  C07C229/12 ,  C07C271/20 ,  C07C2601/02 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32

摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel, trialkyl, 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.

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三烷基，阳离子脂质和核酸 - 脂质颗粒，并且将体内核酸包被的核酸有效地递送到体内。 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。

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。