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2. 发明申请

WO2015069586A2 DUAL MOLECULAR DELIVERY OF OLIGONUCLEOTIDES AND PEPTIDE CONTAINING CONJUGATES 审中-公开
标题翻译：寡分子和含有肽的双分子递送
公开(公告)号：WO2015069586A2
公开(公告)日：2015-05-14
申请号：PCT/US2014063621
申请日：2014-11-03
申请人： MERCK SHARP & DOHME , COLLETTI STEVEN L , TUCKER THOMAS J , TELLERS DAVID M , KIM BOYOUNG , BURKE ROB , CALATI KATHLEEN B , STANTON MATTHEW G , PARMAR RUBINA G , AARONSON JEFFREY G , WANG WEIMIN
发明人： COLLETTI STEVEN L , TUCKER THOMAS J , TELLERS DAVID M , KIM BOYOUNG , BURKE ROB , CALATI KATHLEEN B , STANTON MATTHEW G , PARMAR RUBINA G , AARONSON JEFFREY G , WANG WEIMIN
IPC分类号： A61K48/00
CPC分类号： A61K31/7125 , A61K31/713 , A61K38/10 , A61K38/16 , C07K2319/10 , C12N15/111 , C12N15/113 , C12N15/87 , C12N2310/14 , C12N2310/351 , C12N2320/31
摘要： Disclosed herein is a method for inhibiting expression of a gene of a subject comprising administering (1) a composition comprising R-(L)a-(G)b; wherein R is an oligonucleotide selected from the group consisting of DNA, RNA, siRNA, and microRNA; L is a linker and each occurance of L is independently selected from Table 3; G is a targeting ligand and each occurance of G is independently selected from Table 4; each of a and b is independently 0, 1, 2, 3 or 4; and (2) a composition comprising (P)c-(L)d-(G)e; wherein P is a peptide and each occurance of P is independently selected from Table 2; L is a linker and each occurance of L is independently selected from Table 3; G is a targeting ligand and each occurance of G is independently selected from Table 4; d is 0, 1, 2, 3, 4, 5 or 6; and each of c and e is independently 1, 2, 3, 4, 5 or 6. Compositions in (1) and (2) can be co-administered or sequentially administered.
摘要翻译：本文公开了一种抑制受试者基因表达的方法，包括给予（1）包含R-（L）a-（G）b; 其中R是选自DNA，RNA，siRNA和微小RNA的寡核苷酸; L是连接体，L的每个出现独立地选自表3; G是靶向配体，G的每个出现独立地选自表4; a和b各自独立地为0,1,2,3或4; 和（2）包含（P）c-（L）d-（G）e的组合物; 其中P是肽，并且每个出现的P独立地选自表2; L是连接体，L的每个出现独立地选自表3; G是靶向配体，G的每个出现独立地选自表4; d为0,1,2,3,4,5或6; c和e中的每一个独立地为1,2,3,4,5或6.（1）和（2）中的组合物可以共同施用或依次施用。

3. 发明申请

WO2013016057A1 POLY(ORNITHINE) HOMOPOLYMERS FOR THE DELIVERY OF OLIGONUCLEOTIDES 审中-公开
标题翻译： POLY（ORNITHINE）共聚物用于交付寡核苷酸
公开(公告)号：WO2013016057A1
公开(公告)日：2013-01-31
申请号：PCT/US2012/046989
申请日：2012-07-17
申请人： MERCK SHARP & DOHME CORP. , BARRETT, Stephanie, E. , GARBACCIO, Robert, M. , KOWTONIUK, Robert, A. , WANG, Weimin , WILLIAMS, J. Michael , LIAO, Jing , PEI, Tao , TOBIAS, Sandra, C.
发明人： BARRETT, Stephanie, E. , GARBACCIO, Robert, M. , KOWTONIUK, Robert, A. , WANG, Weimin , WILLIAMS, J. Michael , LIAO, Jing , PEI, Tao , TOBIAS, Sandra, C.
IPC分类号： A61K48/00 , C12Q1/68 , C12P19/34
CPC分类号： C12N15/87 , A61K47/645 , C08G69/10 , C08G69/48 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/344 , C12N2320/32 , C12N2310/317 , C12N2310/332 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The present invention provides poly(amide) polymers, polyconjugates, compositions and methods for the delivery of oligonucleotides for therapeutic purposes.
摘要翻译：本发明提供用于递送用于治疗目的的寡核苷酸的聚（酰胺）聚合物，多偶联物，组合物和方法。

4. 发明申请

WO2011022460A8 NOVEL CATIONIC LIPIDS WITH VARIOUS HEAD GROUPS FOR OLIGONUCLEOTIDE DELIVERY 审中-公开
标题翻译：具有各种头发交付的各种头巾组
公开(公告)号：WO2011022460A8
公开(公告)日：2012-05-10
申请号：PCT/US2010045854
申请日：2010-08-18
申请人： MERCK SHARP & DOHME , CAMERON MARK , DAVIS JENNIFER R , GEISER ANDREA R , STANTON MATTHEW G , TELYATNIKOV VLADISLAV V , TIAN LU , WANG WEIMIN
发明人： CAMERON MARK , DAVIS JENNIFER R , GEISER ANDREA R , STANTON MATTHEW G , TELYATNIKOV VLADISLAV V , TIAN LU , WANG WEIMIN
IPC分类号： C07C229/00 , A61K9/127
CPC分类号： A61K9/1272 , A61K31/7088 , C07J41/0055 , C07J43/003
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with siRNA, to facilitate the cellular uptake and endosomal escape, and to knockdown target mRNA both in vitro and in vivo.
摘要翻译：本发明提供了可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与siRNA形成脂质纳米颗粒以促进细胞摄取和内体逃逸以及在体外和体内。

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

WO2012068187A1 POLY(AMIDE) POLYMERS FOR THE DELIVERY OF OLIGONUCLEOTIDES 审中-公开
标题翻译：用于交付寡核苷酸的聚（酰胺）聚合物
公开(公告)号：WO2012068187A1
公开(公告)日：2012-05-24
申请号：PCT/US2011/060893
申请日：2011-11-16
申请人： MERCK SHARP & DOHME CORP. , BARRETT, Stephanie, E. , BUSUEK, Marina , COLLETTI, Steven, L. , GARBACCIO, Robert, M. , GUIDRY, Erin, N. , KOWTONIUK, Robert, A. , LIAO, Jing , PARISH, Craig, A. , PARMAR, Rubina, G. , PEI, Tao , SCHLOSSER, Kevin, M. , TELLERS, David, M. , TOBIAS, Sandra, C. , TRUONG, Quang, T. , WALDMAN, Jacob, H. , WANG, Weimin , WILLIAMS, J. Michael
发明人： BARRETT, Stephanie, E. , BUSUEK, Marina , COLLETTI, Steven, L. , GARBACCIO, Robert, M. , GUIDRY, Erin, N. , KOWTONIUK, Robert, A. , LIAO, Jing , PARISH, Craig, A. , PARMAR, Rubina, G. , PEI, Tao , SCHLOSSER, Kevin, M. , TELLERS, David, M. , TOBIAS, Sandra, C. , TRUONG, Quang, T. , WALDMAN, Jacob, H. , WANG, Weimin , WILLIAMS, J. Michael
IPC分类号： A61K31/7088
CPC分类号： A61K47/48092 , A61K47/549 , C08G69/10 , C08G73/028 , C08L79/02
摘要： The present invention provides poly(amide) polymers, polyconjugates, compositions and methods for the delivery of oligonucleotides for therapeutic purposes.
摘要翻译：本发明提供用于递送用于治疗目的的寡核苷酸的聚（酰胺）聚合物，多偶联物，组合物和方法。

7. 发明申请

WO2011043913A2 NOVEL CATIONIC LIPIDS WITH SHORT LIPID CHAINS FOR OLIGONUCLEOTIDE DELIVERY 审中-公开
标题翻译：用于低分子量交付的短链脂肪酸的新型阳离子脂肪
公开(公告)号：WO2011043913A2
公开(公告)日：2011-04-14
申请号：PCT/US2010/049420
申请日：2010-09-20
申请人： MERCK SHARP & DOHME CORP. , CAMERON, Mark , DAVIS, Jennifer, R. , WANG, Weimin
发明人： CAMERON, Mark , DAVIS, Jennifer, R. , WANG, Weimin
IPC分类号： A61K48/00 , C07J43/00
CPC分类号： C07J41/0055 , A61K48/0025 , C07J43/003 , C12N15/111 , C12N2310/14 , C12N2310/141 , C12N2320/32
摘要： The instant invention provides for novel cationic lipids with short lipid chains that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides, to facilitate the cellular uptake and endosomal escape, and to knockdown target mRNA both in vitro and in vivo while decreasing inflammatory toxicities.
摘要翻译：本发明提供了具有短脂链的新型阳离子脂质，其可以与其它脂质组分如胆固醇和PEG-脂质组合使用以与寡核苷酸形成脂质纳米颗粒，以促进细胞摄取和内体逃逸，以及敲低靶mRNA 在体内和体内同时减少炎症毒性。

8. 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).

9. 发明申请

WO2005041859A2 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：WO2005041859A2
公开(公告)日：2005-05-12
申请号：PCT/US2004/013456
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： A61K
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 , Y02A50/393 , Y02A50/465 , C12N2310/3521
摘要： This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including cholesterol, folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HSA) 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，包括胆固醇，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（HSA）衍生的生物活性化合物包括抗体，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，同源酶，反义，dsRNA，siNA，siRNA，三重寡核苷酸，2,5-A 嵌合体，诱饵和适体。

10. 发明申请

WO2013166155A1 NOVEL TETRAGALNAC AND PEPTIDE CONTAINING CONJUGATES AND METHODS FOR DELIVERY OF OLIGONUCLEOTIDES 审中-公开
标题翻译：含有结合物的新型四氯化碳和肽以及交付寡核苷酸的方法
公开(公告)号：WO2013166155A1
公开(公告)日：2013-11-07
申请号：PCT/US2013/039072
申请日：2013-05-01
申请人： MERCK SHARP & DOHME CORP. , TELLERS, David , COLLETTI, Steven, L. , DUDKIN, Vadim , AARONSON, Jeffrey , MOMOSE, Aaron , TUCKER, Thomas, Joseph , YUAN, Yu , CALATI, Kathleen, B. , TIAN, Lu , PARMAR, Rubina, G. , SHAW, Anthony, W. , WANG, Weimin , STORR, Rachel, Anne , BUSUEK, Marina , KOWTONIUK, Robert, A.
发明人： TELLERS, David , COLLETTI, Steven, L. , DUDKIN, Vadim , AARONSON, Jeffrey , MOMOSE, Aaron , TUCKER, Thomas, Joseph , YUAN, Yu , CALATI, Kathleen, B. , TIAN, Lu , PARMAR, Rubina, G. , SHAW, Anthony, W. , WANG, Weimin , STORR, Rachel, Anne , BUSUEK, Marina , KOWTONIUK, Robert, A.
IPC分类号： C07H19/056
CPC分类号： C07H15/26 , A61K31/7056 , A61K31/713 , A61K47/549 , C12N15/113 , C12N2310/17 , C12N2310/3513 , A61K2300/00
摘要： Disclosed herein is a modular composition comprising 1) an oligonucleotide; 2) one or more tetraGalNAc ligands of Formula (I), which may be the same or different; optionally, 3) one or more linkers, which may be the same or different; 4) one or more peptides independently selected from Table 3, which may be the same or different; and optionally, 5) one or more targeting ligands, solubilizing agents, pharmacokinetics enhancing agents, lipids, and/or masking agents.
摘要翻译：本文公开了一种模块化组合物，其包含：1）寡核苷酸; 2）一种或多种式（I）的四加仑配体，其可以相同或不同; 任选地，3）一个或多个可以相同或不同的接头; 4）一个或多个独立地选自表3的肽，其可以相同或不同; 和任选地，5）一种或多种靶向配体，增溶剂，药代动力学增强剂，脂质和/或掩蔽剂。

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