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1. 发明申请

WO2008011431A2 RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译：干扰素介导的枯草杆菌蛋白酶KEXIN 9（PCSK9）基因表达的短干扰核酸（SINA）抑制作用
公开(公告)号：WO2008011431A2
公开(公告)日：2008-01-24
申请号：PCT/US2007/073723
申请日：2007-07-17
申请人： SIRNA THERAPEUTICS INC. , MCSWIGGEN, James , JADHAV, Vasant , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
发明人： MCSWIGGEN, James , JADHAV, Vasant , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
IPC分类号： C12N15/11
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/3521
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro- RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases, traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits that are associated with gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对Prop蛋白转化酶枯草杆菌蛋白酶9（PCSK9）基因表达的调节作出响应的性状，疾病和病症的化合物，组合物和方法，和 /或活动。本发明还涉及涉及对Prop蛋白转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达途径或其他细胞过程中涉及的基因的表达和/或活性的调节作出响应的性状，疾病和病症的化合物，组合物和方法调解这些特征，疾病和状况的维持或发展。具体而言，本发明涉及双链核酸分子，包括小核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和能够介导针对Proprotein转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括此类小核酸分子的混合物和此类小核酸分子的脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源RNA分子（例如内源性微RNA（miRNA）（例如，miRNA抑制剂）或内源性短干扰RNA（例如，miRNA））的功能的小核酸分子，例如siNA，siRNA， siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂），通过干扰与内源RNA（例如RISC）相关的内源RNA或蛋白质的调节功能来调节基因表达，，包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子可用于例如提供组合物以预防，抑制或减少代谢疾病，性状和病症，包括但不限于高脂血症，高胆固醇血症，心血管疾病，动脉粥样硬化，高血压，糖尿病（例如类型 I型和/或II型糖尿病），胰岛素抵抗，肥胖和/或与受试者或生物体中的基因表达或活性相关的其他疾病状态，病症或性状。

2. 发明申请

WO2008011467A2 RNA INTERFERENCE MEDIATED INHIBITION OF STEROL REGULATORY ELEMENT-BINDING PROTEIN 1 (SREBP1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的抑制异丙肾上腺素调节蛋白1（SREBP1）的基因表达使用短暂的干扰核酸（SINA）
公开(公告)号：WO2008011467A2
公开(公告)日：2008-01-24
申请号：PCT/US2007/073792
申请日：2007-07-18
申请人： SIRNA THERAPEUTICS INC. , MCSWIGGEN, James , VASANT, Jadhav , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
发明人： MCSWIGGEN, James , VASANT, Jadhav , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
IPC分类号： C12N11/00
CPC分类号： C12N15/1136 , C12N15/113 , C12N2310/14 , C12P19/34
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Sterol Regulatory Element-Binding Protein 1 (SREBP1) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in Sterol Regulatory Element-Binding Protein 1 (SREBP1) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Sterol Regulatory Element-Binding Protein 1 (SREBP1) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate SREBP1 gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits associated with SREBP1 gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对Sterol调控元件结合蛋白1（SREBP1）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。本发明还涉及与涉及Sterol调控元件结合蛋白1（SREBP1）基因表达途径或其他的基因的表达和/或活性的调节作用的性状，疾病和病症相关的化合物，组合物和方法介导这种性状，疾病和状况的维持或发展的细胞过程。具体地说，本发明涉及包括小核酸分子如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和双链RNA 能够介导针对Sterol调节元件结合蛋白1（SREBP1）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸分子的这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）。本发明还涉及可以抑制内源性微RNA（miRNA）（例如miRNA抑制剂）或内源性短干扰RNA（例如miRNA抑制剂）的内源性RNA分子的功能的小核酸分子，例如siNA，siRNA等， siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂）通过干扰与这些内源性RNA相关的内源性RNA或蛋白质的调节功能来调节SREBP1基因表达（例如，RISC ），包括这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子并且可用于例如提供预防，抑制或降低代谢性疾病性状和病症的组合物，包括但不限于高脂血症，高胆固醇血症，心血管疾病，动脉粥样硬化，高血压，糖尿病（例如I型和/或II型糖尿病），与受试者或生物体中的SREBP1基因表达或活性相关的胰岛素抵抗，肥胖症和/或其它疾病状态，病症或性状。

3. 发明申请

WO2007076328A2 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制HEPATITIS C病毒（HCV）基因表达使用短暂的干扰核酸（siNA）
公开(公告)号：WO2007076328A2
公开(公告)日：2007-07-05
申请号：PCT/US2006/062252
申请日：2006-12-18
申请人： SIRNA THERAPEUTICS, INC. , McSWIGGEN, James , MORRISSEY, David , GUERCIOLINI, Roberto , VARGEESE, Chandra , JADHAV, Vasant
发明人： McSWIGGEN, James , MORRISSEY, David , GUERCIOLINI, Roberto , VARGEESE, Chandra , JADHAV, Vasant
IPC分类号： C12N15/11 , A61K31/713 , A61K9/127
CPC分类号： C12N15/1131 , A61K31/713 , A61K47/6911 , C12N15/111 , C12N2310/14 , C12N2310/318 , C12N2310/51 , C12N2770/24211
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticlc (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对基因表达和/或活性调节作出反应的性状，疾病和病症的化合物，组合物和方法。本发明还涉及与涉及参与基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症相关的化合物，组合物和方法，或调节其维持或发展的其他细胞过程性状，疾病和状况。具体地说，本发明涉及包括小核酸分子如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和双链RNA 能够介导针对基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸分子的这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源性微RNA（miRNA）（例如miRNA抑制剂）或内源性短干扰RNA（例如miRNA抑制剂）的内源性RNA分子的功能的小核酸分子，例如siNA，siRNA等， siRNA）（例如siRNA抑制剂）或可以通过干扰与这些内源性RNA（例如RISC）相关的内源性RNA或蛋白质的调节功能来调节基因表达来调节基因表达（例如，RISC抑制剂），包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子并且可用于例如提供组合物以预防，抑制或减少与受试者或生物体中的基因表达或活性相关的疾病，性状和病症。

4. 发明申请

WO2006128141A2 RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的阻断细胞衍生因子-1（SDF-1）基因表达使用短暂的干扰核酸（SINA）
公开(公告)号：WO2006128141A2
公开(公告)日：2006-11-30
申请号：PCT/US2006/020846
申请日：2006-05-26
申请人： SIRNA THERAPEUTICS, INC. , GUERCIOLINI, Roberto , MCSWIGGEN, James , JADHAV, Vasant , VARGEESE, Chandra
发明人： GUERCIOLINI, Roberto , MCSWIGGEN, James , JADHAV, Vasant , VARGEESE, Chandra
IPC分类号： C12N15/113
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3521
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of stromal cell-derived factor- 1 (SDF-I) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in stromal cell-derived factor- 1 (SDF-I) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against stromal cell- derived factor- 1 (SDF-I) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate SDF-I gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce angiogenesis related diseases traits and conditions, including but not limited to ocular disease, cancer and proliferative diseases, traits, and conditions, and/or other disease states, conditions, or traits associated with SDF-I gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对基质细胞衍生因子-1（SDF-1）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。本发明还涉及涉及对参与基质细胞衍生的因子-1（SDF-1）基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症有关的化合物，组合物和方法或调节维持或发展这些性状，疾病和病症的其他细胞过程。具体地说，本发明涉及包括小核酸分子如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和双链RNA 能够介导针对基质细胞衍生因子-1（SDF-1）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸的这种小核酸分子和脂质纳米颗粒（LNP）制剂的混合物酸分子。本发明还涉及可以抑制内源性微RNA（miRNA）（例如miRNA抑制剂）或内源性短干扰RNA（例如miRNA抑制剂）的内源性RNA分子的功能的小核酸分子，例如siNA，siRNA等， siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂）通过干扰与这些内源性RNA相关的内源性RNA或蛋白质的调节功能来调节SDF-1基因表达（例如，，RISC），包括这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子可用于例如提供预防，抑制或减少血管生成相关疾病性状和病症的组合物，包括但不限于眼部疾病，癌症和增殖性疾病，性状和病症，和/或与受试者或生物体中的SDF-1基因表达或活性相关的其它疾病状态，病症或性状。

5. 发明申请

WO2007067981A2 RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR AND VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的血管内皮生长因子和血管内皮生长因子受体基因表达的抑制使用短暂的核酸（siNA）
公开(公告)号：WO2007067981A2
公开(公告)日：2007-06-14
申请号：PCT/US2006/061797
申请日：2006-12-08
申请人： SIRNA THERAPEUTICS, INC. , MCSWIGGEN, James , GUERCIOLINI, Roberto , PAVCO, Pamela
发明人： MCSWIGGEN, James , GUERCIOLINI, Roberto , PAVCO, Pamela
IPC分类号： C12N15/11 , A61K31/713 , A61P27/02
CPC分类号： C12N15/1138 , C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/332 , C12N2320/32
摘要： This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of VEGF and/or VEGFR gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of VEGF and/or VEGFR genes. The application also relates to methods of treating diseases and conditions associated with VEGF and/or VEGFR gene expression, such as ocular diseases and conditions, including age related macular degeneration (AMD) and diabetic retinopathy, as well as providing dosing regimens and treatment protocols.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节VEGF和/或VEGFR基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子调节参与VEGF和/或VEGFR基因表达和/或活性的其它基因的表达和活性的化合物，组合物和方法，所述基因表达和/或活性通过RNA干扰（RNAi）。特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA））分子和用于调节VEGF和/或VEGFR基因表达的方法。该应用还涉及治疗与VEGF和/或VEGFR基因表达相关的疾病和病症的方法，例如眼部疾病和病症，包括年龄相关性黄斑变性（AMD）和糖尿病性视网膜病变，以及提供给药方案和治疗方案。

6. 发明申请

WO2009131661A2 METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF HEPATITIS C VIRUS (HCV) BY DOUBLE-STRANDED RNA 审中-公开
标题翻译：用双重条纹RNA特异性抑制丙型肝炎病毒（HCV）的方法和组合物
公开(公告)号：WO2009131661A2
公开(公告)日：2009-10-29
申请号：PCT/US2009/002470
申请日：2009-04-21
申请人： DICERNA PHARMACEUTICALS, INC. , BEHLKE, Mark, A. , GUERCIOLINI, Roberto , PEEK, Andrew, S.
发明人： BEHLKE, Mark, A. , GUERCIOLINI, Roberto , PEEK, Andrew, S.
IPC分类号： C12P21/06
CPC分类号： C12N15/1131 , C12N2310/14

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