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1. 发明申请

WO2010129672A1 LIPOPHILIC POLYNUCLEOTIDE CONJUGATES 审中-公开
标题翻译：脂多糖共混物
公开(公告)号：WO2010129672A1
公开(公告)日：2010-11-11
申请号：PCT/US2010/033729
申请日：2010-05-05
申请人： MIRAGEN THERAPEUTICS , MARSHALL, William S. , VAGLE, Kurt
发明人： MARSHALL, William S. , VAGLE, Kurt
IPC分类号： C07H21/00
CPC分类号： C07J41/0055 , A61K48/0033 , C07J43/003 , C07J51/00 , C12N15/111 , C12N15/113 , C12N15/87 , C12N2310/113 , C12N2310/315 , C12N2310/3515
摘要： Disclosed are lipophilic polynucleotide conjugates including polynucleotide-cholesterol conjugates and methods of delivering therapeutic polynucleotides to a mammalian cell or patient in need of treatment using said conjugates. The disclosure further provides methods of synthesizing the lipophilic polynucleotide conjugates.The conjugates are designed to mimic or target cellular miRNAs. The lipophilic moiety, such as cholesterol or cholesterol derivative, is spaced from the polynucleotide by a substantially linear hydrocarbon group. Due to an absence of significantly polar groups and/or exchangeable protons in the vicinity of the lipophilic moiety, the interaction between the lipophilic moiety and cell membranes is enhanced to provide for efficient entry into cells.
摘要翻译：公开了亲脂性多核苷酸缀合物，包括多核苷酸 - 胆固醇缀合物和将治疗性多核苷酸递送至需要使用所述缀合物治疗的哺乳动物细胞或患者的方法。本公开进一步提供了合成亲脂性多核苷酸缀合物的方法。设计缀合物以模拟或靶向细胞miRNA。亲脂性部分，例如胆固醇或胆固醇衍生物，通过基本上线性的烃基与多核苷酸间隔开。由于在亲油部分附近不存在显着的极性基团和/或可交换的质子，增强了亲脂部分和细胞膜之间的相互作用以提供有效进入细胞。

2. 发明申请

WO2012061810A1 BASE MODIFIED OLIGONUCLEOTIDES 审中-公开
标题翻译：基因修饰寡核苷酸
公开(公告)号：WO2012061810A1
公开(公告)日：2012-05-10
申请号：PCT/US2011/059588
申请日：2011-11-07
申请人： MIRAGEN THERAPEUTICS , VAGLE, Kurt , DALBY, Christina , MARSHALL, William S.
发明人： VAGLE, Kurt , DALBY, Christina , MARSHALL, William S.
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/141 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/33 , C12N2310/3521 , C12N2310/3523 , C12N2310/3525 , C12N2310/3527 , C12N2310/3529 , C12N2320/52
摘要： The present invention relates to oligonucleotides with base modified nucleosides for enhancement of binding affinity.
摘要翻译：本发明涉及具有用于增强结合亲和力的碱修饰核苷的寡核苷酸。

3. 发明申请

WO2008147438A3 LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE MOLECULES 审中-公开
公开(公告)号：WO2008147438A3
公开(公告)日：2008-12-04
申请号：PCT/US2007/081594
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： C12N15/88 , A61K47/48 , A61K9/127
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).

4. 发明申请

WO2007086881A2 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：WO2007086881A2
公开(公告)日：2007-08-02
申请号：PCT/US2006/005127
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
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

WO2007086883A2 LIPID NANOPARTICLE BASED COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOGICALLY 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：WO2007086883A2
公开(公告)日：2007-08-02
申请号：PCT/US2006/005311
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
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）。

6. 发明申请

WO2015142735A1 SYNTHESIS OF BICYCLIC NUCLEOSIDES 审中-公开
标题翻译：双相核苷酸的合成
公开(公告)号：WO2015142735A1
公开(公告)日：2015-09-24
申请号：PCT/US2015/020761
申请日：2015-03-16
申请人： MIRAGEN THERAPEUTICS, INC.
发明人： VAGLE, Kurt
IPC分类号： C07H21/04 , C12P19/30 , C12P19/38
CPC分类号： C07H19/16 , C07H1/00 , C07H19/06 , C07H19/073 , C07H19/10 , C07H19/173 , C12P19/30 , C12P19/38
摘要： The present disclosure provides methods for synthesizing modified nucleosides, nucleotides, and oligonucleotides comprising at least one 2'-C-Bridged Bicyclic Nucleotide, and to intermediates used in the process.
摘要翻译：本公开提供了用于合成包含至少一个2'-C-桥连双环核苷酸的修饰的核苷，核苷酸和寡核苷酸的方法，以及用于该方法的中间体。

7. 发明申请

WO2014145356A1 BRIDGED BICYCLIC NUCLEOSIDES 审中-公开
标题翻译：桥接双相核素
公开(公告)号：WO2014145356A1
公开(公告)日：2014-09-18
申请号：PCT/US2014/030100
申请日：2014-03-16
申请人： MIRAGEN THERAPEUTICS, INC.
发明人： VAGLE, Kurt
IPC分类号： C12N15/11 , C07H21/04
CPC分类号： C12N15/113 , C07H19/10 , C07H21/04 , C12N15/111 , C12N2310/113 , C12N2310/315 , C12N2310/3231 , C12N2310/346 , C12N2320/50 , C12N2330/30
摘要： The present invention relates to 2'-C-Bridged Bicyclic Nucleosides and Nucleotides, and oligonucleotides comprising at least one 2'-C-Bridged Bicyclic Nucleotides. The present invention further provides pharmaceutical compositions comprising the nucleosides, nucleotides, and oligonucleotides, as well as their respective methods of use and synthesis.
摘要翻译：本发明涉及2'-C-桥连双环核苷和核苷酸，以及包含至少一个2'-C-桥连双环核苷酸的寡核苷酸。本发明还提供包含核苷，核苷酸和寡核苷酸的药物组合物，以及它们各自的使用和合成方法。

8. 发明申请

WO2020181107A1 MICRORNA MIMICS AND USES THEREOF 审中-公开
公开(公告)号：WO2020181107A1
公开(公告)日：2020-09-10
申请号：PCT/US2020/021223
申请日：2020-03-05
申请人： MIRAGEN THERAPEUTICS, INC.
发明人： VAGLE, Kurt , DALBY, Christina Marie , ROY, Subhadeep
IPC分类号： C12N15/113 , A61K31/713 , A61K31/7105
摘要： Provided herein are synthetic, modified miRNA mimics that increase miRNA activity. In particular, the present disclosure provides synthetic, modified mimics of miR-29, miR-96, miR-182, and miR-183, as well as methods of making and uses thereof.

9. 发明公开

EP2970968A1 BRIDGED BICYCLIC NUCLEOSIDES 有权
标题翻译：桥接的双环核苷
公开(公告)号：EP2970968A1
公开(公告)日：2016-01-20
申请号：EP14765516.1
申请日：2014-03-16
申请人： Miragen Therapeutics, Inc.
发明人： VAGLE, Kurt
IPC分类号： C12N15/11 , C07H21/04
CPC分类号： C12N15/113 , C07H19/10 , C07H21/04 , C12N15/111 , C12N2310/113 , C12N2310/315 , C12N2310/3231 , C12N2310/346 , C12N2320/50 , C12N2330/30
摘要： The present invention relates to 2′-C-Bridged Bicyclic Nucleosides and Nucleotides, and oligonucleotides comprising at least one 2′-C-Bridged Bicyclic Nucleotides. The present invention further provides pharmaceutical compositions comprising the nucleosides, nucleotides, and oligonucleotides, as well as their respective methods of use and synthesis.
摘要翻译：本发明涉及2'-C-桥连双环核苷和核苷酸，以及包含至少一个2'-C-桥连双环核苷酸的寡核苷酸。本发明进一步提供了包含核苷，核苷酸和寡核苷酸的药物组合物，以及它们各自的使用和合成方法。

10. 发明申请

WO2020181130A2 POLYNUCLEOTIDE CONJUGATES AND USES THEREOF 审中-公开
公开(公告)号：WO2020181130A2
公开(公告)日：2020-09-10
申请号：PCT/US2020/021255
申请日：2020-03-05
申请人： MIRAGEN THERAPEUTICS, INC.
发明人： ROY, Subhadeep , VAGLE, Kurt , RIGBY, Kevin , MONTGOMERY, Rusty L.
IPC分类号： C12N15/113
摘要： Provided herein are polynucleotide conjugates comprising a polynucleotide and an agent, wherein the polynucleotide comprises a synthetic double stranded miR-29 mimic and the agent facilitates the delivery of the polynucleotide to a cell type or tissue type involved in fibrosis or inflammation. Also provided are methods of making, and uses thereof.

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