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    Compositions and Methods for Inhibiting Expression of a Target Gene
    2.
    发明申请
    Compositions and Methods for Inhibiting Expression of a Target Gene 有权
    抑制靶基因表达的组合物和方法

    公开(公告)号：US20150353937A1

    公开(公告)日：2015-12-10

    申请号：US14737304

    申请日：2015-06-11

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1138 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/50

    摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

    摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。

    Compositions and Methods for Inhibiting Expression of a Target Gene
    3.
    发明申请
    Compositions and Methods for Inhibiting Expression of a Target Gene 有权
    抑制靶基因表达的组合物和方法

    公开(公告)号：US20140106450A1

    公开(公告)日：2014-04-17

    申请号：US14012994

    申请日：2013-08-28

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1138 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/50

    摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

    摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。

    Compositions and methods for inhibiting expression of a target gene
    6.
    发明授权
    Compositions and methods for inhibiting expression of a target gene 有权
    用于抑制靶基因表达的组合物和方法

    公开(公告)号：US09587240B2

    公开(公告)日：2017-03-07

    申请号：US14737304

    申请日：2015-06-11

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    IPC分类号： A61K31/713 ,  A61K31/7115 ,  C12N15/113 ,  C12N15/63 ,  C12N15/11 ,  A61K38/00

    CPC分类号： C12N15/1138 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/50

    摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

    摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。

    Compositions and methods for inhibiting expression of a target gene
    7.
    发明授权
    Compositions and methods for inhibiting expression of a target gene 有权
    用于抑制靶基因表达的组合物和方法

    公开(公告)号：US09074213B2

    公开(公告)日：2015-07-07

    申请号：US14012994

    申请日：2013-08-28

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Roland Kreutzer ,  Stefan Limmer ,  Sylvia Limmer ,  Philipp Hadwiger

    IPC分类号： C12N15/113 ,  C12N15/11 ,  A61K38/00

    CPC分类号： C12N15/1138 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2320/50

    摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.

    摘要翻译： 本发明涉及具有与靶基因的至少一部分基本上相同且长度不超过49个，优选小于25个核苷酸的核苷酸序列的双链核糖核酸（dsRNA），其中 包含在3'末端具有1至4个核苷酸突出端和5'-末端的互补（反义）RNA链。 本发明还涉及包含dsRNA和药学上可接受的载体的药物组合物。 药物组合物可用于抑制靶基因的表达，以及用于治疗由靶基因表达引起的疾病，以低剂量（即，小于5毫克，优选小于25微克，每kg体重/ 天）。 本发明还涉及抑制靶基因表达的方法，以及用于治疗由基因表达引起的疾病的方法。