公开(公告)号：US20120244210A1

公开(公告)日：2012-09-27

申请号：US13437428

申请日：2012-04-02

申请人： Fumikazu SHINOHARA ,  Tetsuo Yoshida ,  Hiroko Sugishita ,  Tomoyuki Naoi ,  Toshihiko Ishii ,  Junichi Enokizono

发明人： Fumikazu SHINOHARA ,  Tetsuo Yoshida ,  Hiroko Sugishita ,  Tomoyuki Naoi ,  Toshihiko Ishii ,  Junichi Enokizono

IPC分类号： A61K9/127 ,  A61P35/00 ,  A61P29/00 ,  A61K31/713

CPC分类号： A61K9/0019 ,  A61K9/1271 ,  C12N15/1135 ,  C12N15/88 ,  C12N2310/14 ,  C12N2320/32

摘要： The present invention provides a composition that comprises a lipidparticle encapsulating a double-stranded nucleic acid molecule, wherein the antisense strand is a polynucleotide of 17 to 30 bases in which a sequence is complementary to the sequence of the 17 contiguous bases of a target gene's mRNA, the sense strand is a polynucleotide of 17 to 30 bases that contains a base sequence complementary to the base sequence of bases 1 to 17 in the 5′-end to 3′-end direction of the antisense strand, and a particular amount of the sugars binding to certain bases of the antisense strand and the sense strand are deoxyribose, or ribose whose hydroxyl group at the 2′ position is substituted by a modifying group, and wherein the lipidparticle contains a lipid bilayer membrane whose constituent component is a lipid conjugate, a fatty acid conjugate or an aliphatic hydrocarbon conjugate of a water-soluble substance.

摘要翻译： 本发明提供了包含包封双链核酸分子的脂质粒子的组合物，其中，所述反义链是17〜30个碱基的多核苷酸，其中序列与靶基因的mRNA的17个连续碱基的序列互补 有义链是17〜30个碱基的多核苷酸，其含有与反义链的5'端至3'-端方向的碱基1至17的碱基序列互补的碱基序列，特定量的 与反义链和有义链的某些碱基结合的糖是脱氧核糖或其2'位的羟基被修饰基团取代的核糖，并且其中所述脂质颗粒含有脂质双层膜，其构成成分是脂质缀合物， 脂肪酸结合物或水溶性物质的脂族烃共轭物。

公开(公告)号：US09061042B2

公开(公告)日：2015-06-23

申请号：US13017562

申请日：2011-01-31

申请人： Nobuhiro Yagi ,  Junichi Enokizono

发明人： Nobuhiro Yagi ,  Junichi Enokizono

IPC分类号： C12N15/11 ,  A61K31/7105 ,  A61K9/00 ,  A61K9/127 ,  A61K31/712 ,  A61K31/713 ,  C12N15/113

CPC分类号： A61K31/7105 ,  A61K9/0019 ,  A61K9/1271 ,  A61K31/712 ,  A61K31/713 ,  C12N15/1135 ,  C12N2310/14 ,  Y10T436/143333

摘要： An object of the present invention is to provide a composition for suppressing expression of a target gene, and the like. A composition comprising an RNA-encapsulated liposome, wherein the RNA contains a sequence consisting of 15 to 30 contiguous bases of a target gene mRNA (hereinafter, sequence X) and a base sequence (hereinafter, complementary sequence X′) complementary to the sequence X, 1 to 90% of all sugars binding to the bases of the sequence X and the complementary sequence X′ being ribose substituted by a modifying group at 2′ position, and the lipid particle being capable of reaching a tissue or an organ containing an expression site of the target gene is provide.

摘要翻译： 本发明的目的是提供抑制靶基因表达的组合物等。 包含RNA包封的脂质体的组合物，其中所述RNA含有由靶基因mRNA（以下称为序列X）的15〜30个连续碱基和与序列X互补的碱基序列（以下称为互补序列X'）组成的序列 ，与所述序列X的碱基结合的所有糖的1至90％，并且所述互补序列X'是在2'位置被修饰基团取代的核糖，并且所述脂质颗粒能够到达包含表达的组织或器官 提供靶基因的位点。

公开(公告)号：US20140039034A1

公开(公告)日：2014-02-06

申请号：US13980139

申请日：2011-02-02

申请人： Fumikazu Shinohara ,  Tetsuo Yoshida ,  Hiroko Sugishita ,  Tomoyuki Naoi ,  Toshihiko Ishii ,  Junichi Enokizono

发明人： Fumikazu Shinohara ,  Tetsuo Yoshida ,  Hiroko Sugishita ,  Tomoyuki Naoi ,  Toshihiko Ishii ,  Junichi Enokizono

IPC分类号： A61K47/44

CPC分类号： A61K47/44 ,  A61K9/1272 ,  A61K31/713 ,  C12N15/113 ,  C12N15/1136 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2320/32

摘要： The present invention provides a composition that comprises a lipidparticle encapsulating a double-stranded nucleic acid molecule, wherein the antisense strand is a polynucleotide of 17 to 30 bases in which a sequence is complementary to the sequence of the 17 contiguous bases of a target gene's mRNA, the sense strand is a polynucleotide of 17 to 30 bases that contains a base sequence complementary to the base sequence of bases 1 to 17 in the 5′-end to 3′-end direction of the antisense strand, and a particular amount of the sugars binding to certain bases of the antisense strand and the sense strand are deoxyribose, or ribose whose hydroxyl group at the 2′ position is substituted by a modifying group, and wherein the lipidparticle contains a lipid bilayer membrane whose constituent component is a lipid conjugate, a fatty acid conjugate or an aliphatic hydrocarbon conjugate of a water-soluble substance.

摘要翻译： 本发明提供了包含包封双链核酸分子的脂质粒子的组合物，其中，所述反义链是17〜30个碱基的多核苷酸，其中序列与靶基因的mRNA的17个连续碱基的序列互补 有义链是17〜30个碱基的多核苷酸，其含有与反义链的5'端至3'-端方向的碱基1至17的碱基序列互补的碱基序列，特定量的 与反义链和有义链的某些碱基结合的糖是脱氧核糖或其2'位的羟基被修饰基团取代的核糖，并且其中所述脂质颗粒含有脂质双层膜，其构成成分是脂质缀合物， 脂肪酸结合物或水溶性物质的脂族烃共轭物。

公开(公告)号：US20110182980A1

公开(公告)日：2011-07-28

申请号：US13017562

申请日：2011-01-31

申请人： Nobuhiro Yagi ,  Junichi Enokizono

发明人： Nobuhiro Yagi ,  Junichi Enokizono

IPC分类号： A61K9/127 ,  A61K31/713 ,  A61K9/50 ,  G01N33/53 ,  A61P35/00 ,  A61P29/00

CPC分类号： A61K31/7105 ,  A61K9/0019 ,  A61K9/1271 ,  A61K31/712 ,  A61K31/713 ,  C12N15/1135 ,  C12N2310/14 ,  Y10T436/143333

摘要： An object of the present invention is to provide a composition for suppressing expression of a target gene, and the like. A composition comprising an RNA-encapsulated liposome, wherein the RNA contains a sequence consisting of 15 to 30 contiguous bases of a target gene mRNA (hereinafter, sequence X) and a base sequence (hereinafter, complementary sequence X′) complementary to the sequence X, 1 to 90% of all sugars binding to the bases of the sequence X and the complementary sequence X′ being ribose substituted by a modifying group at 2′ position, and the lipid particle being capable of reaching a tissue or an organ containing an expression site of the target gene is provide.

摘要翻译： 本发明的目的是提供抑制靶基因表达的组合物等。 包含RNA包封的脂质体的组合物，其中所述RNA含有由靶基因mRNA（以下称为序列X）的15〜30个连续碱基和与序列X互补的碱基序列（以下称为互补序列X'）组成的序列 ，与所述序列X的碱基结合的所有糖的1至90％，并且所述互补序列X'是在2'位置被修饰基团取代的核糖，并且所述脂质颗粒能够到达包含表达的组织或器官 提供靶基因的位点。

公开(公告)号：US20120207818A1

公开(公告)日：2012-08-16

申请号：US13348959

申请日：2012-01-12

申请人： Fumikazu SHINOHARA ,  Tetsuo Yoshida ,  Hiroko Sugishita ,  Tomoyuki Naoi ,  Toshihiko Ishii ,  Junichi Enokizono

发明人： Fumikazu SHINOHARA ,  Tetsuo Yoshida ,  Hiroko Sugishita ,  Tomoyuki Naoi ,  Toshihiko Ishii ,  Junichi Enokizono

IPC分类号： A61K9/127 ,  A61P35/00 ,  A61P29/00 ,  A61K31/7088

CPC分类号： A61K31/713 ,  A61K9/0019 ,  A61K9/10 ,  A61K9/1271 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2320/32 ,  C12N2310/321 ,  C12N2310/3521

摘要： The present invention provides a composition that comprises a liposome encapsulating a double-stranded nucleic acid molecule, wherein the antisense strand is a polynucleotide of 17 to 30 bases in which a sequence is complementary to the sequence of the 17 contiguous bases of a target gene's mRNA, the sense strand is a polynucleotide of 17 to 30 bases that contains a base sequence complementary to the base sequence of bases 1 to 17 in the 5′-end to 3′-end direction of the antisense strand, and a particular amount of the sugars binding to certain bases of the antisense strand and the sense strand are deoxyribose, or ribose whose hydroxyl group at the 2′ position is substituted by a modifying group, and wherein the liposome contains a lipid bilayer membrane whose constituent component is a lipid conjugate, a fatty acid conjugate or an aliphatic hydrocarbon conjugate of a water-soluble substance.

摘要翻译： 本发明提供一种包含包封双链核酸分子的脂质体的组合物，其中，所述反义链是17〜30个碱基的多核苷酸，其中序列与靶基因的mRNA的17个连续碱基的序列互补 有义链是17〜30个碱基的多核苷酸，其含有与反义链的5'端至3'-端方向的碱基1至17的碱基序列互补的碱基序列，特定量的 与反义链和有义链的某些碱基结合的糖是脱氧核糖或其2'位的羟基被修饰基团取代的核糖，并且其中所述脂质体含有脂质双层膜，其构成成分是脂质缀合物， 脂肪酸结合物或水溶性物质的脂族烃共轭物。