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    RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE ( PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    1.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE ( PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
    使用新浪环核苷酸磷酸二酯酶4型（PDE4B）的基因表达的RNA干扰介导的抑制（短干扰核酸）

    公开(公告)号：EP2152877A2

    公开(公告)日：2010-02-17

    申请号：EP08747579.4

    申请日：2008-05-02

    申请人： Sirna Therapeutics Inc.

    发明人： STRAPPS, Walter ,  JADHAV, Vasant ,  VARGEESE, Chandra ,  RICHARDS, Ivan

    IPC分类号： C12N15/11

    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.

    LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE MOLECULES
    2.
    发明公开
    LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE MOLECULES 审中-公开
    ON脂质纳米粒基组合物和用于将生物活性分子的方法

    公开(公告)号：EP2104740A2

    公开(公告)日：2009-09-30

    申请号：EP07873552.9

    申请日：2007-10-17

    申请人： Sirna Therapeutics, Inc.

    发明人： CHEN, Tongqian ,  VARGEESE, Chandra ,  VAGLE, Kurt ,  WANG, Weimin ,  ZHANG, Ye

    IPC分类号： C12N15/88 ,  A61K47/48 ,  A61K9/127

    CPC分类号： C12N15/88 ,  A61K9/1271 ,  A61K47/6911 ,  C07C217/28 ,  Y10S977/797

    摘要： The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double- stranded RNA (dsRNA), micro-RNA (miRNA), short hairpin RNA (shRNA), and RNAi inhibitor molecules, to relevant cells and/or tissues, such as in a subject or organism. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).

    Conjugates and compositions for cellular delivery
    5.
    发明公开
    Conjugates and compositions for cellular delivery 有权
    Konjugate und Zusammensetzungen zurzellulärenVerabreichung

    公开(公告)号：EP2415486A2

    公开(公告)日：2012-02-08

    申请号：EP11184529.3

    申请日：2002-05-17

    申请人： Sirna Therapeutics, Inc.

    发明人： BEIGELMAN, Leonid ,  MATULIC-ADAMIC, Jasenka ,  VARGEESE, Chandra ,  KARPEISKY, Alexander ,  SHAFFER, Christopher ,  BLATT, Lawrence

    IPC分类号： A61K47/48 ,  A61K9/14 ,  C08G63/08

    CPC分类号： B82Y5/00 ,  A61K47/544 ,  A61K47/549 ,  A61K47/551 ,  A61K47/60 ,  A61K47/66

    摘要： This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HAS) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones nucleosides, nucleotide, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers.

    摘要翻译： 本发明的特征在于缀合物，可降解接头，组合物，合成方法及其应用，包括叶酸，半乳糖，半乳糖胺，N-乙酰半乳糖胺，PEG，磷脂，肽和人血清白蛋白（HAS）衍生的生物活性化合物的缀合物，包括 抗体，抗病毒剂，化学治疗剂，肽，蛋白质，激素核苷，核苷酸，非核苷和核酸，包括酶核酸，DNA酶，异构酶，反义，dsRNA，siRNA，三重寡核苷酸，2,5-A嵌合体，诱饵和适体 。

    RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    6.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
    RNA干扰介导的环核苷酸4型磷酸二酯酶（PDE4B）基因表达的短期干扰核酸（SINA）抑制作用

    公开(公告)号：EP2152875A2

    公开(公告)日：2010-02-17

    申请号：EP08747557.0

    申请日：2008-05-02

    申请人： Sirna Therapeutics Inc.

    发明人： STRAPPS, Walter ,  JADHAV, Vasant ,  VARGEESE, Chandra ,  RICHARDS, Ivan

    IPC分类号： C12N15/11

    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-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，诊断和治疗对环核苷酸4型磷酸二酯酶（PDE4B）基因表达和/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，包括PDE4B1，PDE4B2 ，和PDE4B3基因表达和/或活性。 本发明还涉及与响应参与环核苷酸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/或活性的调节有关的性状，疾病和病症的化合物，组合物和方法 其介导了包括但不限于IL-6，IL-1，IL-8，IL-15，TNF-α和基质金属蛋白酶（MMP）如MMP-1在内的这些性状，疾病和病症的维持或发展 ，MMP-2，MMP-3，MMP-9和MMP-12。特别地，本发明涉及双链核酸分子，包括小核酸分子，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），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RNA分子或RNAi途径组分​​（RNAi抑制剂）的功能的小核酸分子，例如siNA，siRNA，反义和其它，例如内源性微RNA（miRNA）（例如， 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（例如RISC抑制剂）通过干扰这种内源性RNA或蛋白质的调节功能来调节PDE4B基因​​表达 与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）制剂的内源RNA（例如RISC）相关。 例如，这种小核酸分子可用于提供组合物以预防，抑制或减轻与受试者中的PDE4B基因​​表达或活性相关的炎症，呼吸和自身免疫疾病，性状和/或病症和/或其他疾病状态 或有机体。

    RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    7.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
    通过RNA干扰介导的抑制前蛋白转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达的使用短干扰核酸（siNA）

    公开(公告)号：EP2052079A2

    公开(公告)日：2009-04-29

    申请号：EP07813032.5

    申请日：2007-07-17

    申请人： Sirna Therapeutics Inc.

    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.

    RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    8.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
    通过RNA干扰介导的来源的间质的基因的表达抑制因子1使用短干扰核酸（SDF-1）（新浪网）

    公开(公告)号：EP1891217A2

    公开(公告)日：2008-02-27

    申请号：EP06784507.3

    申请日：2006-05-26

    申请人： Sirna Therapeutics, Inc.

    发明人： GUERCIOLINI, Roberto ,  MCSWIGGEN, James ,  JADHAV, Vasant ,  VARGEESE, Chandra

    IPC分类号： C12N15/11 ,  C07K14/52

    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.