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61. 发明申请

WO2005016355A1 INTRAVASCULAR DELIVERY OF NON-VIRAL NUCLEIC ACID 审中-公开
标题翻译：非病毒性肝炎的血管内输送
公开(公告)号：WO2005016355A1
公开(公告)日：2005-02-24
申请号：PCT/US2003/035460
申请日：2003-11-05
申请人： MIRUS CORPORATION
发明人： WOLFF, Jon, A. , HAGSTROM, James, E. , BUDKER, Vladimir, G. , ROZEMA, David, B. , MONAHAN, Sean, D. , SLATTUM, Paul, M.
IPC分类号： A61K31/70
CPC分类号： A61K48/005 , A61K31/7088 , A61K38/1808 , A61K38/1825 , A61K47/59 , A61K47/62 , A61K47/645 , A61K48/0008 , A61K48/0016 , A61K48/0025 , A61K48/0041 , A61K48/0075 , A61K48/0083 , C07H21/00 , A61K2300/00
摘要： Disclosed is a process for delivering genetic material into a mammalian cell to alter endogenous properties of the cell. The process comprises designing a polynucleotide for transfection. Then the polynucleotide is inserted into a mammalian vessel such as an artery. Prior to insertion, subsequent to insertion, or concurrent with insertion the permeability of the vessel is increased thereby the genetic material is delivered to the parenchymal cell altering endogenous properties of the cell.
摘要翻译：公开了将遗传物质递送到哺乳动物细胞中以改变细胞的内源性质的方法。该方法包括设计用于转染的多核苷酸。然后将多核苷酸插入哺乳动物血管如动脉。在插入之前，随后插入或同时插入，血管的渗透性增加，从而将遗传物质递送至实质细胞，从而改变细胞的内源性质。

62. 发明申请

WO2004058308A1 AN EFFICIENT NON-VIRAL GENE/DRUG DELIVERY SYSTEM 审中-公开
标题翻译：有效的非病毒基因/药物递送系统
公开(公告)号：WO2004058308A1
公开(公告)日：2004-07-15
申请号：PCT/US2003/040893
申请日：2003-12-22
申请人： BOARD OF REGENTS THE UNIVERSITY OF TEXAS SYSTEM , ZOU, Yiyu
发明人： ZOU, Yiyu
IPC分类号： A61K48/00
CPC分类号： A61K48/0041 , A61K47/62 , A61K47/6911 , A61K48/0025
摘要： The present invention provides novel compositions for delivering macromolecules and micromolecules to a cell. The present invention is non-viral system and comprises formulations of lipids and polymers that are able to efficiently and safely deliver a molecule as a therapeutic modality to a subject.
摘要翻译：本发明提供用于将大分子和小分子递送到细胞的新型组合物。本发明是非病毒系统，并且包括能够有效和安全地将分子作为治疗方式递送给受试者的脂质和聚合物制剂。

63. 发明申请

WO2003097805A2 DELIVERY OF NUCLEIC ACID-LIKE COMPOUNDS 审中-公开
标题翻译：交付核酸类化合物
公开(公告)号：WO2003097805A2
公开(公告)日：2003-11-27
申请号：PCT/US2003/015378
申请日：2003-05-15
申请人： CALIFORNIA PACIFIC MEDICAL CENTER
发明人： HONG, Keelung , ZHENG, Wei-Wen , DRUMMOND, Daryl, C. , KIRPOTIN, Dmitri, B. , HAYES, Mark, Eamon
IPC分类号： C12N
CPC分类号： A61K9/1272 , A61K9/127 , A61K9/1271 , A61K31/711 , A61K48/0025 , A61K48/0041 , A61K48/0091
摘要： A process for preparing a microparticulate complex is provided. The process comprises: a) combining a particle-forming component ("PFC") and a nucleic acid-like component ("NAC") in a monophasic composition comprising water and a water-miscible, organic solvent to form a mixture wherein the PFC and the NAC are independently molecularly or micellarly soluble in the aqueous/organic solvent system, and b) reducing the amount of the organic solvent in the mixture. This effects formation of the microparticulate complex of the NAC and the PFC. Also provided is a microparticulate complex that comprises a particle-forming component complexed to a nucleic acid-like component forming an approximately spherical particle, wherein the particle-forming component encloses an interior of the particle containing the nucleic acid-like component and the so-enclosed interior volume has less than about 50 % (preferably less than 20 %) of the volume containing free water. Also disclosed composition comprising water and particles of the microparticulate complex. According to the invention a nucleic acid-like is component is delivered to a cell by (a) contacting the cell with a composition comprising water and the microparticulate complex, and (b) maintaining the contact for a time sufficient to allow the nucleic acid-based moiety to enter the cell. A therapeutic nucleic acid-like component is delivered into a patient in need thereof by administering a composition comprising water and particles of the microparticulate complex. Also disclosed is a charge-changing composition represented by the formula A-X-B, wherein X represents a chemical bond capable of irreversible dissociation in reaction to a factor in a physiological or bioprocess environment; A represents a molecular moiety that upon dissociation of the bond X produces a ionically charged product; and B represents a molecular moiety, which upon the dissociation of bond X, separates from the composition leaving the remaining ionically charged product more positive than that of A-X-B itself.
摘要翻译：提供了一种制备微粒复合物的方法。该方法包括：a）在包含水和水混溶性有机溶剂的单相组合物中组合形成颗粒的组分（“PFC”）和核酸样组分（“NAC”）以形成混合物，其中PFC 并且NAC独立地在水/有机溶剂体系中分子或胶束溶解，和b）减少混合物中有机溶剂的量。这会影响NAC和PFC的微粒复合物的形成。还提供了一种微粒复合物，其包含与形成近似球形颗粒的核酸样成分复合的颗粒形成组分，其中所述颗粒形成组分包含含有核酸样组分的颗粒的内部，封闭的内部容积小于含有游离水的体积的约50％（优选小于20％）。还公开了包含水和微粒复合物颗粒的组合物。根据本发明，通过（a）使细胞与包含水和微粒复合物的组合物接触，将核酸样的成分递送至细胞，和（b）将接触保持足以允许核酸 - 基部分进入细胞。治疗性核酸样成分通过施用包含水和微粒复合物颗粒的组合物递送到有需要的患者中。还公开了由式A-X-B表示的电荷变化组合物，其中X表示在与生理或生物过程环境中的因素反应时能够不可逆解离的化学键; A表示分子部分，其在键X解离时产生离子带电荷的产物; 并且B表示分子部分，其在键X的解离时与组合物分离，留下剩余的带电荷的产物比A-X-B本身的阳离子更正。

64. 发明申请

WO2003094971A1 CATIONIC LIPIDS FOR INTRACELLULAR DELIVERY OF BIOACTIVE SUBSTANCES 审中-公开
标题翻译：用于细胞内递送生物物质的阳离子脂肪
公开(公告)号：WO2003094971A1
公开(公告)日：2003-11-20
申请号：PCT/SG2003/000109
申请日：2003-05-10
申请人： JOHNS HOPKINS SINGAPORE PTE LTD , LEONG, Kam , JIE, Wen , MAO, Hai, Quan , WANG, Jun
发明人： LEONG, Kam , JIE, Wen , MAO, Hai, Quan , WANG, Jun
IPC分类号： A61K47/18
CPC分类号： A61K48/0025 , A61K9/1272 , C07J51/00 , C12N15/88
摘要： The present invention provides biodegradable cationic liposomes of a mixture of a cationic lipid and a neutral lipid, cationic liposome compositions, and methods of using same for the controlled release of a biologically active substance to a specified tissue or cells. Preferred cationic lipids for use in cationic liposomes include cationic lipids having a positively charged group and two hydroxyl groups which are capable of complexing one or more biologically active substances. Preferred methods include the controlled release of biologically active substances and gene therapy using cationic liposomes and compositions composed thereof.
摘要翻译：本发明提供了阳离子脂质和中性脂质的混合物的可生物降解的阳离子脂质体，阳离子脂质体组合物，以及将其用于控制释放生物活性物质到指定组织或细胞的方法。用于阳离子脂质体的优选阳离子脂质包括具有带正电基团的阳离子脂质和能够络合一种或多种生物活性物质的两个羟基。优选的方法包括使用阳离子脂质体及其组成的组合物的生物活性物质的控制释放和基因治疗。

65. 发明申请

WO02088318A3 LIPID-COMPRISING DRUG DELIVERY COMPLEXES AND METHODS FOR THEIR PRODUCTION 审中-公开
标题翻译：脂肪包裹药物输送复合物及其生产方法
公开(公告)号：WO02088318A3
公开(公告)日：2003-05-15
申请号：PCT/US0213609
申请日：2002-04-30
申请人： TARGETED GENETICS CORP , EMERALD GENE SYSTEMS LTD , HARVIE PIERROT , PAUL RALPH , CUDMORE SALLY , O'MAHONY DANIEL J
发明人： HARVIE PIERROT , PAUL RALPH , CUDMORE SALLY , O'MAHONY DANIEL J
IPC分类号： C12N15/09 , A61K9/127 , A61K47/22 , A61K47/24 , A61K47/28 , A61K47/32 , A61K47/34 , A61K47/42 , A61K47/46 , A61K47/48 , A61K48/00 , A61P29/00 , A61P43/00 , C07K5/09 , C07K7/08 , C07K9/00 , C07K14/025 , C07K14/59 , C07K14/62 , C12N15/88 , C12N15/00 , C12N15/63
CPC分类号： C12N15/88 , A61K9/1272 , A61K47/58 , A61K47/60 , A61K48/0025 , C12N2810/40 , C12N2810/405 , C12N2810/854
摘要： Novel stable, concentrated, biologically active and ready-to-use lipid-comprising drug delivery complexes and methods for their production are described. The complexes of the invention comprise a drug, at least one lipid species, optionally at least one polycation, and at least one targeting factor. The at least one lipid species may comprise a pegylated lipid. The complexes of the invention may provoke lower levels of inflammatory cytokines such as tumor necrosis factor-alpha (TNF-alpha). The method described herein provides for the large scale production of lipid-comprising drug delivery systems useful for gene therapy and other applications.
摘要翻译：描述了新型稳定的，浓缩的，生物活性的和即用型含脂质的药物递送复合物及其生产方法。本发明的复合物包含药物，至少一种脂质物质，任选的至少一种聚阳离子和至少一种靶向因子。所述至少一种脂质物质可以包含聚乙二醇化的脂质。本发明的复合物可能引起较低水平的炎性细胞因子，例如肿瘤坏死因子-α（TNF-α）。本文所述的方法提供大规模生产用于基因治疗和其它应用的含脂质药物递送系统。

66. 发明申请

WO2003004685A2 SOLUBLE STEROIDAL PEPTIDES FOR NUCLEIC ACID DELIVERY 审中-公开
标题翻译：用于核酸递送的可溶性甾体肽
公开(公告)号：WO2003004685A2
公开(公告)日：2003-01-16
申请号：PCT/US2002/021265
申请日：2002-07-03
申请人： UNIVERSITY OF UTAH RESEARCH FOUNDATION , MAHATO, Ram, I. , MAHESHWARI, Anurag , KIM, Sung, Wan
发明人： MAHATO, Ram, I. , MAHESHWARI, Anurag , KIM, Sung, Wan
IPC分类号： C12Q
CPC分类号： A61K38/208 , A61K38/10 , A61K47/554 , A61K48/00 , A61K48/0025 , C07K14/47 , C07K14/5434 , C12N15/87
摘要： Amphiphilic lipopeptide compositions for gene delivery are disclosed. An illustrative amphiphilic lipopeptide composition includes a human protamine 2 peptide conjugated to a hydrophobic moiety. Illustrative hydrophobic moieties include sterols, bile acids, and fatty acids. The amphiphilic lipopeptide composition is mixed with a nucleic acid such that the nucleic acid binds to the peptide portion of the lipopeptide. This mixture is placed in contact with mammalian cells to effect transfection of the cells with the nucleic acid. A method of making such amphiphilic lipopeptides is also described.
摘要翻译：公开了用于基因递送的两亲性脂肽组合物。示例性的两性脂肽组合物包括缀合至疏水部分的人鱼精蛋白2肽。说明性疏水部分包括甾醇，胆汁酸和脂肪酸。将两亲性脂肽组合物与核酸混合，使得核酸结合脂肽的肽部分。将该混合物与哺乳动物细胞接触以使核酸转染细胞。还描述了制备这种两亲性脂肽的方法。

67. 发明申请

WO2003002598A2 PEPTIDES FOR USE AS TRANSLOCATION FACTORS 审中-公开
标题翻译：用作移位因子的肽
公开(公告)号：WO2003002598A2
公开(公告)日：2003-01-09
申请号：PCT/GB2002/003027
申请日：2002-07-01
申请人： IMPLYX LTD. , CRISANTI, Andrea
发明人： CRISANTI, Andrea
IPC分类号： C07K14/00
CPC分类号： A61K38/08 , A61K8/64 , A61K47/64 , A61K48/0025 , A61K2800/57 , A61Q17/04 , A61Q19/00 , C07K2319/10 , C12N15/62
摘要： Proteins that contain the amino acid sequence motif X 1 X 1 X 2 X 3 X 1 , where X 1 = R or K and X 2 and X 3 = any amino acid have been found to translocate and can therefore be used in the manufacture of compositions for therapeutic applications. The proteins may also be used as translocation factors to deliver proteins or nucleic acids into a cell.
摘要翻译：含有氨基酸序列基序X 1 X X 1 X 2 X X 3的蛋白质，其中X 1 = R或K和X 2和X 3 =任何氨基酸已被发现易位并因此可用于制造用于治疗应用的组合物。蛋白质也可以作为易位因子将蛋白质或核酸输送到细胞中。

68. 发明申请

WO2002059261A2 INHIBITION OF RESTENOSIS USING A DNA-COATED STENT 审中-公开
标题翻译：使用DNA涂层的静脉抑制保留
公开(公告)号：WO2002059261A2
公开(公告)日：2002-08-01
申请号：PCT/US2001/045755
申请日：2001-12-07
申请人： THE MEDSTAR RESEARCH INSTITUTE , EPSTEIN, Stephen, E. , FUCHS, Shmuel
发明人： EPSTEIN, Stephen, E. , FUCHS, Shmuel
IPC分类号： C12N
CPC分类号： A61K48/0025 , A61K47/6957 , A61K48/00 , A61L31/005 , A61L31/10 , A61L31/16 , A61L2300/258 , A61L2300/416 , A61L2300/64 , C07K14/52 , C07K14/78 , C12N9/6435 , C12Y304/21007 , C08L89/06
摘要： Restenosis of arteries after angioplasty is inhibited by implanting in the treated artery a stent incorporating genes that encode gene products having anti-restenotic activity. The genes may be incorporated into a coating on the stent structure or in cells that are affixed to the stent. The genes or cells containing them may be adhered to the struts of the stent or incorporated in a collagen matrix that forms a coating covering the struts and interstices of the stent
摘要翻译：通过在处理的动脉中植入具有编码具有抗再狭窄活性的基因产物的基因（106）的支架（100）来抑制血管成形术后动脉的再狭窄。基因（106）可以结合到支架结构上的涂层（108）中，或者固定在支架（100）上的细胞（114）中。含有它们的基因（106）或细胞（114）可以粘附到支架（100）的支柱（102）上，或者结合到形成覆盖支柱（102）和间隙（104）的涂层（110）的胶原基质中）的支架（100）。

69. 发明申请

WO02030957A1 PEPTIDE-BASED GEMINI SURFACTANT COMPOUNDS FACILITATING THE TRANSFER INTO CELLS 审中-公开
标题翻译：基于肽的GEMINI表面活性剂化合物促进细胞转移
公开(公告)号：WO02030957A1
公开(公告)日：2002-04-18
申请号：PCT/GB2001/004529
申请日：2001-10-11
IPC分类号： C12N15/09 , A61K31/7088 , A61K38/00 , A61K47/24 , A61K47/34 , A61K47/42 , A61K48/00 , A61P43/00 , C07K5/02 , C07K7/02 , C07K7/06 , C12N15/87 , C07K5/10 , C12N15/67
CPC分类号： A61K48/0025 , C07K5/0215 , C07K7/02 , C12N15/87
摘要： Peptide-based gemini compounds comprising basic amino acid chains linked by at least epsilon amide bond, showing improved DNA transfection properties, are disclosed. Methods for production of the compounds and the uses thereof are also disclosed.
摘要翻译：公开了包含通过至少ε-酰胺键连接的碱性氨基酸链的基于肽的双体组合物，显示出改进的DNA转染性质。还公开了化合物的制备方法及其用途。

70. 发明申请

WO02010197A1 CATIONIC AMPHIPATHIC PEPTIDES AND THE APPLICATION THEREOF IN GENE TRANSFER VECTORS 审中-公开
标题翻译：阳离子磷脂肽及其在基因转移载体中的应用
公开(公告)号：WO02010197A1
公开(公告)日：2002-02-07
申请号：PCT/ES2001/000274
申请日：2001-07-06
IPC分类号： A61K48/00 , C07K14/47 , C12N15/87 , C07K14/00
CPC分类号： C12N15/87 , A61K48/0025 , C07K14/4723
摘要： Said peptides comprise residues of basic aliphatic and aromatic amino acids on both ends of the peptide and, preferably, an aromatic amino acid residue in position 3 of the peptide sequence. These peptides have simultaneously the capacity of binding a polynucleotide and disrupting biological membranes. Said peptides are useful in the construction of non-viral gene transfer vectors with improved gene material condensing capacity and/or reduced cytotoxicity and/or improved genetic material transfer efficiency. The invention can be used in gene transfer for research and therapeutic purposes.
摘要翻译：所述肽包含肽两端的碱性脂族和芳族氨基酸的残基，优选肽序列的第3位的芳香族氨基酸残基。这些肽同时具有结合多核苷酸并破坏生物膜的能力。所述肽可用于构建具有改善的基因材料冷凝能力和/或降低的细胞毒性和/或改善的遗传物质转移效率的非病毒基因转移载体。本发明可用于基因转移用于研究和治疗目的。

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