公开(公告)号：US20100063308A1

公开(公告)日：2010-03-11

申请号：US12617079

申请日：2009-11-12

申请人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

IPC分类号： C07C229/00

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）。

公开(公告)号：US07641915B2

公开(公告)日：2010-01-05

申请号：US12048023

申请日：2008-03-13

申请人： TongQian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  WeiMin Wang ,  Ye Zhang

发明人： TongQian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  WeiMin Wang ,  Ye Zhang

IPC分类号： A61K9/127 ,  A61K31/56

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）。

公开(公告)号：US20090048197A1

公开(公告)日：2009-02-19

申请号：US12027952

申请日：2008-02-07

申请人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

IPC分类号： A61K31/7088 ,  C07H21/04 ,  A61P43/00

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 相关细胞的酸分子，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）。

公开(公告)号：US20100048888A1

公开(公告)日：2010-02-25

申请号：US12446882

申请日：2007-10-17

申请人： Tongqian Chen ,  Kurt Vagle ,  Chandra Vargeese ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Kurt Vagle ,  Chandra Vargeese ,  Weimin Wang ,  Ye Zhang

IPC分类号： C07J43/00

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 相关细胞的酸分子，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）。

公开(公告)号：US07514099B2

公开(公告)日：2009-04-07

申请号：US11353630

申请日：2006-02-14

申请人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

IPC分类号： A61K9/127 ,  A61K31/56

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）。

公开(公告)号：US20110092739A1

公开(公告)日：2011-04-21

申请号：US12973238

申请日：2010-12-20

申请人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

IPC分类号： C07C211/27

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）。

公开(公告)号：US20100105933A1

公开(公告)日：2010-04-29

申请号：US12640342

申请日：2009-12-17

申请人： Tongqian Chen ,  Kurt Vagle ,  Chandra Vargeese ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Kurt Vagle ,  Chandra Vargeese ,  Weimin Wang ,  Ye Zhang

IPC分类号： C07J9/00

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 相关细胞的酸分子，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）。

公开(公告)号：US07691405B2

公开(公告)日：2010-04-06

申请号：US12027952

申请日：2008-02-07

申请人： Tongqian Chen ,  Kurt Vagle ,  Chandra Vargeese ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Kurt Vagle ,  Chandra Vargeese ,  Weimin Wang ,  Ye Zhang

IPC分类号： A61K9/127 ,  A61K51/00 ,  C07H21/04

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. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver short interfering nucleic acid (siNA). 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。 具体地，本发明涉及有效转染或递送短干扰核酸（siNA）的新型阳离子脂质，微粒，纳米颗粒和转染试剂。 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）。

公开(公告)号：US07404969B2

公开(公告)日：2008-07-29

申请号：US11586102

申请日：2006-10-24

申请人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

IPC分类号： A61K9/127 ,  A61K51/00 ,  C07H21/04

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. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver short interfering nucleic acid (siNA). 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。 具体地，本发明涉及有效转染或递送短干扰核酸（siNA）的新型阳离子脂质，微粒，纳米颗粒和转染试剂。 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）。

公开(公告)号：US20060240554A1

公开(公告)日：2006-10-26

申请号：US11353630

申请日：2006-02-14

申请人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

发明人： Tongqian Chen ,  Chandra Vargeese ,  Kurt Vagle ,  Weimin Wang ,  Ye Zhang

IPC分类号： C12N5/02 ,  C07C229/10

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).