专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

WO2005041859A3 CONJUGATES AND COMPOSITIONS FOR CELLULAR DELIVERY. 审中-公开
标题翻译：细胞传递的结合物和组合物。
公开(公告)号：WO2005041859A3
公开(公告)日：2007-04-26
申请号：PCT/US2004013456
申请日：2004-04-30
申请人： SIRNA THERAPEUTICS INC , VARGEESE CHANDRA , HAEBERLI PETER , WANG WEIMIN , CHEN TONGQIAN
发明人： VARGEESE CHANDRA , HAEBERLI PETER , WANG WEIMIN , CHEN TONGQIAN
IPC分类号： A61K31/7105 , A61K31/713 , A61K38/00 , A61K47/48 , C07H17/00 , C07H17/02 , C07H21/02 , C07H21/04 , C12N1/15 , C12N5/02 , C12N5/22 , C12N15/113 , C12N15/12 , C12N15/51 , C12N15/88
CPC分类号： C12N15/1138 , A61K38/00 , A61K47/544 , A61K47/549 , A61K47/551 , A61K47/554 , A61K47/60 , A61K47/64 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12Y604/01002 , C12N2310/3521
摘要： This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including cholesterol, galactosamine, and N-acetyl galactosamine derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones, nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siNA, 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，包括生物活性化合物的胆固醇，半乳糖胺和N-乙酰半乳糖胺衍生的缀合物，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，siNA，siRNA，三重寡核苷酸，2,5-A嵌合体，诱饵和适体。

2. 发明申请

WO2007086881A3 CATIONIC LIPIDS AND FORMULATED MOLECULAR COMPOSITIONS CONTAINING THEM 审中-公开
标题翻译：包含它们的阳离子脂肪和配体分子组合物
公开(公告)号：WO2007086881A3
公开(公告)日：2008-02-28
申请号：PCT/US2006005127
申请日：2006-02-14
申请人： SIRNA THERAPEUTICS INC , CHEN TONGQIAN , VARGEESE CHANDRA , VAGLE KURT , WANG WEIMIN , ZHANG YE
发明人： CHEN TONGQIAN , VARGEESE CHANDRA , VAGLE KURT , WANG WEIMIN , ZHANG YE
IPC分类号： C07C217/28 , C07C217/84 , C07J41/00
CPC分类号： C07C217/28 , A61K9/0073 , A61K9/1271 , A61K9/1272 , A61K47/543 , A61K47/6911 , C07C217/42 , C07C217/58 , C07C239/20 , C07J41/00 , C12N15/88
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), and short hairpin RNA (shRNA) 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).
摘要翻译：本发明涉及新型阳离子脂质，转染剂，微粒，纳米颗粒和短干扰核酸（siNA）分子。本发明还涉及用于研究，诊断和治疗对受试者或生物体中基因表达和/或活性的调节作用的性状，疾病和病状的组合物和方法。具体地说，本发明涉及有效转染或递送生物活性分子的新型阳离子脂质，微粒，纳米颗粒和转染剂，例如抗体（例如单克隆抗体，嵌合型，人源化等），胆固醇，激素，抗病毒剂，肽，蛋白质，化学治疗剂，小分子，维生素，辅因子，核苷，核苷酸，寡核苷酸，酶核酸，反义核酸，形成三链体的寡核苷酸，2,5-A嵌合体，dsRNA，同源酶，适体，诱饵及其类似物和小核酸酸分子，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）分子）相关细胞和/或组织，例如受试者或生物体。这样的新型阳离子脂质，微粒，纳米颗粒和转染剂可用于例如提供预防，抑制或治疗细胞，受试者或生物体中的疾病，病症或性状的组合物。本文所述的组合物通常被称为配制的分子组合物（FMC）或脂质纳米颗粒（LNP）。

3. 发明申请

WO2008147438A2 LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE MOLECULES 审中-公开
标题翻译：基于脂质纳米颗粒的组合物和用于递送生物活性分子的方法
公开(公告)号：WO2008147438A2
公开(公告)日：2008-12-04
申请号：PCT/US2007081594
申请日：2007-10-17
申请人： SIRNA THERAPEUTICS INC , CHEN TONGQIAN , VARGEESE CHANDRA , VAGLE KURT , WANG WEIMIN , ZHANG YE
发明人： CHEN TONGQIAN , VARGEESE CHANDRA , VAGLE KURT , WANG WEIMIN , ZHANG YE
IPC分类号： C12N15/87 , A61K9/127 , A61K47/48
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).
摘要翻译：本发明涉及新型阳离子脂质，转染剂，微粒，纳米颗粒和短干扰核酸（siNA）分子。本发明还涉及用于研究，诊断和治疗对受试者或生物体中基因表达和/或活性的调节作用的性状，疾病和病状的组合物和方法。具体地说，本发明涉及有效转染或递送生物活性分子的新型阳离子脂质，微粒，纳米颗粒和转染剂，例如抗体（例如单克隆抗体，嵌合型，人源化等），胆固醇，激素，抗病毒剂，肽，蛋白质，化学治疗剂，小分子，维生素，辅因子，核苷，核苷酸，寡核苷酸，酶核酸，反义核酸，形成三链体的寡核苷酸，2,5-A嵌合体，dsRNA，同源酶，适体，诱饵及其类似物和小核酸酸性分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA），短发夹RNA（shRNA）和RNAi抑制剂分子，和/或组织，例如受试者或生物体。这样的新型阳离子脂质，微粒，纳米颗粒和转染剂可用于例如提供预防，抑制或治疗细胞，受试者或生物体中的疾病，病症或性状的组合物。本文所述的组合物通常被称为配制的分子组合物（FMC）或脂质纳米颗粒（LNP）。

4. 发明申请

WO2007086883A3 CATIONIC LIPIDS AND FORMULATED MOLECULAR COMPOSITIONS CONTAINING THEM 审中-公开
标题翻译：包含它们的阳离子脂肪和配体分子组合物
公开(公告)号：WO2007086883A3
公开(公告)日：2007-12-27
申请号：PCT/US2006005311
申请日：2006-02-14
申请人： SIRNA THERAPEUTICS INC , CHEN TONGQIAN , VARGEESE CHANDRA , VAGLE KURT , WANG WEIMIN , ZHANG YE
发明人： CHEN TONGQIAN , VARGEESE CHANDRA , VAGLE KURT , WANG WEIMIN , ZHANG YE
IPC分类号： C07C217/28 , C07C217/84 , C07J41/00
CPC分类号： C07C217/28 , A61K9/0073 , A61K9/1271 , A61K9/1272 , A61K47/543 , A61K47/6911 , C07C217/42 , C07C217/58 , C07C239/20 , C07J41/00 , C12N15/88
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), and short hairpin RNA (shRNA) 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).
摘要翻译：本发明涉及新型阳离子脂质，转染剂，微粒，纳米颗粒和短干扰核酸（siNA）分子。本发明还涉及用于研究，诊断和治疗对受试者或生物体中基因表达和/或活性的调节作用的性状，疾病和病状的组合物和方法。具体地说，本发明涉及有效转染或递送生物活性分子的新型阳离子脂质，微粒，纳米颗粒和转染剂，例如抗体（例如单克隆抗体，嵌合型，人源化等），胆固醇，激素，抗病毒剂，肽，蛋白质，化学治疗剂，小分子，维生素，辅因子，核苷，核苷酸，寡核苷酸，酶核酸，反义核酸，形成三链体的寡核苷酸，2,5-A嵌合体，dsRNA，同源酶，适体，诱饵及其类似物和小核酸酸分子，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）分子）相关细胞和/或组织，例如受试者或生物体。这样的新型阳离子脂质，微粒，纳米颗粒和转染剂可用于例如提供预防，抑制或治疗细胞，受试者或生物体中的疾病，病症或性状的组合物。本文所述的组合物通常被称为配制的分子组合物（FMC）或脂质纳米颗粒（LNP）。

5. 发明申请

WO2005007854A2 POLYCATIONIC COMPOSITIONS FOR CELLULAR DELIVERY OF POLYNUCLEOTIDES 审中-公开
标题翻译：用于多核苷酸细胞传递的多聚阳离子组合物
公开(公告)号：WO2005007854A2
公开(公告)日：2005-01-27
申请号：PCT/US2004021997
申请日：2004-07-09
申请人： SIRNA THERAPEUTICS INC , VARGEESE CHANDRA , WEIMIN WANG , CHEN TONGQIAN , SWEEDLER DAVID , HAEBERLI PETER
发明人： VARGEESE CHANDRA , WEIMIN WANG , CHEN TONGQIAN , SWEEDLER DAVID , HAEBERLI PETER
IPC分类号： A61K38/00 , A61K47/48 , C12N15/11 , C12N15/113 , C12N15/87
CPC分类号： A61K49/0008 , A61K38/00 , A61K47/645 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N15/1138 , C12N15/87 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/32 , C12N2330/30 , C12Y103/01022 , C12Y604/01002 , C12N2310/3521
摘要： The present invention relates to delivery of biologically active molecules to cells. Specifically, the invention relates to polycationic compositions, polymers and methods for delivering nucleic acids, polynucleotides, and oligonucleotides such RNA, DNA and analogs thereof, including short interfering RNA (siRNA), ribozymes, and antisense, or peptides, polypeptides, proteins, antibodies, hormones and small molecules, to cells by facilitating transport across cellular membranes epithelial tissues and endothelial tissues. The compositions and methods of the invention are useful in therapeutic, research, and diagnostic applications that rely upon the efficient transfer of biologically active molecules into cells, tissues, and organs.
摘要翻译：本发明涉及生物活性分子向细胞的递送。具体而言，本发明涉及用于递送核酸，多核苷酸和寡核苷酸如RNA，DNA及其类似物（包括短干扰RNA（siRNA），核酶和反义或肽，多肽，蛋白质，抗体）的聚阳离子组合物，聚合物和方法，激素和小分子，促进细胞膜上皮组织和内皮组织的转运。本发明的组合物和方法可用于依赖于生物活性分子向细胞，组织和器官的有效转移的治疗，研究和诊断应用。

6. 发明申请

WO2008137751A3 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：WO2008137751A3
公开(公告)日：2009-02-12
申请号：PCT/US2008062512
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： C12N15/113
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），例如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。

7. 发明申请

WO2008137775A2 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：WO2008137775A2
公开(公告)日：2008-11-13
申请号：PCT/US2008062548
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 , C12N15/113
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/53 , C12Y301/04017 , 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。

8. 发明申请

WO2008011467A3 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：利用短干扰核酸（SINA）RNA干扰介导的甾醇调节元件结合蛋白1（SREBP1）基因表达抑制
公开(公告)号：WO2008011467A3
公开(公告)日：2008-08-07
申请号：PCT/US2007073792
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： C12N15/113
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，诊断和治疗对固醇调节元件结合蛋白1（SREBP1）基因表达和/或活性的调节作出反应的性状，疾病和病症的化合物，组合物和方法。本发明还涉及涉及对Sterol调控元件结合蛋白1（SREBP1）基因表达途径或其他基因参与的基因的表达和/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，疾病和病症的维持或发展的细胞过程。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）和能够介导针对固醇调节元件结合蛋白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鸡尾酒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SREBP1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特征和病症，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I型和/或II型糖尿病），胰岛素抵抗，肥胖症和/或与受试者或生物体中的SREBP1基因表达或活性相关的其他疾病状态，病症或性状。

9. 发明申请

WO2007120527A2 RNA INTERFERENCE MEDIATED INHIBITION OF RESPIRATORY SYNCYTIAL VIRUS (RSV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的呼吸道同源性病毒（RSV）表达的抑制
公开(公告)号：WO2007120527A2
公开(公告)日：2007-10-25
申请号：PCT/US2007008186
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： C12N15/113
CPC分类号： C12N15/1131 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/3521
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.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节呼吸道合胞病毒（RSV）基因表达的化合物，组合物和方法。本发明还涉及用于通过使用小核酸分子调节参与RSV基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其他基因的表达和活性的化合物，组合物和方法。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））分子和方法用于调节RSV基因的表达，包括这种小核酸分子的混合物和这种这种小核酸分子的脂质纳米颗粒制剂的混合物。该应用还涉及治疗与RSV基因表达相关的疾病和病症的方法，例如RSV感染，呼吸衰竭，细支气管炎和肺炎，以及提供给药方案和治疗方案。

10. 发明申请

WO2006128141A3 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干扰介导的使用短暂干扰核酸（SINA）的细胞衍生因子-1（SDF-1）基因表达的抑制作用
公开(公告)号：WO2006128141A3
公开(公告)日：2007-07-12
申请号：PCT/US2006020846
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： C07K14/52 , 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，组合物和方法或介导这些性状，疾病和状况的维持或发展的其他细胞过程。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。这样的小核酸分子并且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内的SDF-1基因表达或活性相关的其它疾病状态，病症或性状。

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式