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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

WO2007120527A3 RNA INTERFERENCE MEDIATED INHIBITION OF RESPIRATORY SYNCYTIAL VIRUS (RSV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
公开(公告)号：WO2007120527A3
公开(公告)日：2007-10-25
申请号：PCT/US2007/008186
申请日：2007-03-30
申请人： SIRNA THERAPEUTICS INC. , VAISH, Narendra, K. , VARGEESE, Chandra , MCSWIGGEN, James
发明人： VAISH, Narendra, K. , VARGEESE, Chandra , MCSWIGGEN, James
IPC分类号： C12N15/11
摘要： This invention relates to compounds, compositions, and methods useful for modulating sespiratory syncytial virus (RSV) 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 RSV 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 RSV genes, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such such small nucleic acid molecules cocktails thereof. The application also relates to methods of treating diseases and conditions associated with RSV gene expression, such as RSV infection, respiratory failure, bronchiolitis and pneumonia, as well as providing dosing regimens and treatment protocols.

6. 发明申请

WO2007147143A3 RNA INTERFERENCE MEDIATED INHIBITION OF 11 BETA-HYDROXYSTEROID DEHYDROGENASE-1 (11 BETA-HSD-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
公开(公告)号：WO2007147143A3
公开(公告)日：2007-12-21
申请号：PCT/US2007/071388
申请日：2007-06-15
申请人： SIRNA THERAPEUTICS, INC. , MCSWIGGEN, James , VARGEESE, Chandra , JADHAV, Vasant
发明人： MCSWIGGEN, James , VARGEESE, Chandra , JADHAV, Vasant
IPC分类号： C12N15/11 , C12P19/34 , C07H21/02 , C07H21/04 , A01N43/04
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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-HSD-1 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 diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity, hypercholesterolemia, cardiovascular disease and/or other disease states, conditions, or traits associated with 11 beta-HSD-1 gene expression or activity in a subject or organism.

7. 发明申请

WO2008137761A3 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
公开(公告)号：WO2008137761A3
公开(公告)日：2008-11-13
申请号：PCT/US2008/062530
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/11 , A61K31/713
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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.

8. 发明申请

WO2008137775A3 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2008137775A3
公开(公告)日：2008-11-13
申请号：PCT/US2008/062548
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/11
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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-7, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.

9. 发明申请

WO2008137771A2 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE ( PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制循环核型4型磷酸酯酶（PDE4B）使用短暂干扰核酸（siNA）的基因表达
公开(公告)号：WO2008137771A2
公开(公告)日：2008-11-13
申请号：PCT/US2008/062544
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/09
CPC分类号： C12N15/1137 , A01K2207/05 , A01K2227/105 , A01K2267/0368 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-7, IL-8, IL-15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP-2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗响应于环核苷酸4型磷酸二酯酶（PDE4B）基因表达和/或活性的调节的性状，疾病和病症的化合物，组合物和方法，包括PDE4B1，PDE4B2 ，和PDE4B3基因表达和/或活性。本发明还涉及涉及与参与环核苷酸4型磷酸二酯酶（PDE4B）基因表达途径或其他细胞过程的基因的表达和/或活性的调节相关的性状，疾病和病症的化合物，组合物和方法包括但不限于IL-6，IL-7，IL-8，IL-15，TNF-α和基质金属蛋白酶（MMP），如MMP-1 ，MMP-2，MMP-3，MMP-9和MMP-12。具体而言，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）和短发夹RNA（shRNA），以及能够介导针对环核苷酸4型磷酸二酯酶（PDE4B）基因表达的RNA干扰（RNAi）的多功能siNA分子，包括鸡尾酒的苏 ch这种小核酸分子的小核酸分子和脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源性RNA分子或RNAi途径组分（RNAi抑制剂）如内源性微RNA（miRNA）的功能的小核酸分子，例如siNA，siRNA，反义寡核苷酸和其它， miRNA抑制剂）或内源性短干扰RNA（siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（如RISC抑制剂）调节PDE4B基因表达的干扰该内源性RNA或蛋白质的调控功能与这样的内源性RNA（例如，RISC）相关联，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，抑制或减少与受试者中PDE4B基因表达或活性相关的炎症，呼吸和自身免疫性疾病，性状和病症和/或其它疾病状态的组合物或生物体。

10. 发明申请

WO2008137776A2 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的抑制循环核型4型磷酸酯酶（PDE4B）基因表达使用短暂的核酸（SINA）
公开(公告)号：WO2008137776A2
公开(公告)日：2008-11-13
申请号：PCT/US2008/062549
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/09
CPC分类号： C12N15/1137 , C12N2310/14 , C12Y301/04017
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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗响应于环核苷酸4型磷酸二酯酶（PDE4B）基因表达和/或活性的调节的性状，疾病和病症的化合物，组合物和方法，包括PDE4B1，PDE4B2 ，和PDE4B3基因表达和/或活性。本发明还涉及涉及与参与环核苷酸4型磷酸二酯酶（PDE4B）基因表达途径或其他细胞过程的基因的表达和/或活性的调节相关的性状，疾病和病症的化合物，组合物和方法包括但不限于IL-6，IL-1，IL-8，IL-15，TNF-α和基质金属蛋白酶（MMP），如MMP-1 ，MMP-2，MMP-3，MMP-9和MMP-12。具体地说，本发明涉及包括小核酸分子的双链核酸分子，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），以及能够介导针对环核苷酸4型磷酸二酯酶（PDE4B）基因表达的RNA干扰（RNAi）的多功能siNA分子，包括鸡尾酒的这种小核酸分子的这种小核酸分子和脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源性RNA分子或RNAi途径组分（RNAi抑制剂）如内源性微RNA（miRNA）的功能的小核酸分子，例如siNA，siRNA，反义寡核苷酸和其它， miRNA抑制剂）或内源性短干扰RNA（siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（如RISC抑制剂）调节PDE4B基因表达的干扰该内源性RNA或蛋白质的调控功能与这样的内源性RNA（例如，RISC）相关联，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，抑制或减少与受试者中PDE4B基因表达或活性相关的炎症，呼吸和自身免疫性疾病，性状和病症和/或其它疾病状态的组合物或生物体。

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