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公开(公告)号:US20120027803A1
公开(公告)日:2012-02-02
申请号:US13153200
申请日:2011-06-03
申请人: Muthiah MANOHARAN , Martin Maier , Muthusamy Jayaraman , Shigeo Matsuda , Narayanannair K. Jayaprakash , Kallanthottathil G. Rajeev , Akin Akinc , Thomas A. Baillie
发明人: Muthiah MANOHARAN , Martin Maier , Muthusamy Jayaraman , Shigeo Matsuda , Narayanannair K. Jayaprakash , Kallanthottathil G. Rajeev , Akin Akinc , Thomas A. Baillie
IPC分类号: A61K47/44 , A61K31/713 , A61P37/04 , C12N5/071 , C12N15/85 , C07C229/30 , A61K31/7088
CPC分类号: C07C229/12 , A61K31/713 , A61K39/0005 , A61K47/44 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/30
摘要: The present invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
摘要翻译: 本发明涉及一种阳离子脂质,其具有位于阳离子脂质的脂质部分(例如,疏水链)的中间或远端部分中的一个或多个可生物降解基团。 这些阳离子脂质可以掺入用于递送活性剂(例如核酸)的脂质颗粒中。 本发明还涉及包含中性脂质,能够减少聚集的脂质,本发明的阳离子脂质和任选的固醇的脂质颗粒。 脂质颗粒可以进一步包括治疗剂如核酸。
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公开(公告)号:US09012498B2
公开(公告)日:2015-04-21
申请号:US13153200
申请日:2011-06-03
申请人: Muthiah Manoharan , Martin Maier , Muthusamy Jayaraman , Shigeo Matsuda , Narayanannair K. Jayaprakash , Kallanthottathil G. Rajeev , Akin Akinc , Thomas A. Baillie
发明人: Muthiah Manoharan , Martin Maier , Muthusamy Jayaraman , Shigeo Matsuda , Narayanannair K. Jayaprakash , Kallanthottathil G. Rajeev , Akin Akinc , Thomas A. Baillie
IPC分类号: A61K31/265 , A61K31/13 , C07C229/00 , C07C229/12 , C12N15/88
CPC分类号: C07C229/12 , A61K31/713 , A61K39/0005 , A61K47/44 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/30
摘要: The present invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
摘要翻译: 本发明涉及一种阳离子脂质,其具有位于阳离子脂质的脂质部分(例如,疏水链)的中间或远端部分中的一个或多个可生物降解基团。 这些阳离子脂质可以掺入用于递送活性剂(例如核酸)的脂质颗粒中。 本发明还涉及包含中性脂质,能够减少聚集的脂质,本发明的阳离子脂质和任选的固醇的脂质颗粒。 脂质颗粒可以进一步包括治疗剂如核酸。
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公开(公告)号:US08642076B2
公开(公告)日:2014-02-04
申请号:US13211094
申请日:2011-08-16
申请人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayaraman , Martin Maier
发明人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayaraman , Martin Maier
IPC分类号: A61K9/48
CPC分类号: C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要: Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译: 描述了用于施用基于核酸的疗法,例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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公开(公告)号:US20120046478A1
公开(公告)日:2012-02-23
申请号:US13211094
申请日:2011-08-16
申请人: Muthiah MANOHARAN , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人: Muthiah MANOHARAN , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号: C07C271/16 , C07C235/74 , C07C69/40
CPC分类号: C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要: Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译: 描述了用于施用基于核酸的疗法,例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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公开(公告)号:US08034376B2
公开(公告)日:2011-10-11
申请号:US12056230
申请日:2008-03-26
申请人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号: A61K9/127
CPC分类号: C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要: Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译: 描述了用于施用基于核酸的疗法,例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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公开(公告)号:US20090023673A1
公开(公告)日:2009-01-22
申请号:US12056230
申请日:2008-03-26
申请人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
发明人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayraman , Martin Maier
IPC分类号: A61K47/14 , A61K31/7088
CPC分类号: C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要: Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译: 描述了用于施用基于核酸的疗法,例如缔合复合物如脂质体和脂质复合物的组合物和方法。
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公开(公告)号:US20130017223A1
公开(公告)日:2013-01-17
申请号:US13516335
申请日:2010-12-17
申请人: Michael J Hope , Thomas D. Madden , Pieter R. Cullis , Martin Maier , Muthusamy Jayaraman , Kallanthottathil G. Rajeev , Akin Akinc , Muthiah Manoharan
发明人: Michael J Hope , Thomas D. Madden , Pieter R. Cullis , Martin Maier , Muthusamy Jayaraman , Kallanthottathil G. Rajeev , Akin Akinc , Muthiah Manoharan
IPC分类号: A61K47/24 , A61K47/28 , A61P37/04 , A61K31/713 , C12N5/071 , C12N15/85 , A61K31/7088 , A61K31/7105
CPC分类号: A61K47/543 , A61K9/1272 , A61K47/6911 , C12N15/88
摘要: A lipid particle can include a plurality of cationic lipids, such as a first cationic lipid and a second cationic lipid. The first cationic lipid can be selected on the basis of a first property and the second cationic can be selected on the basis of a second property. The first and second properties are complementary. The attributes of the lipid particle can reflect the selected properties of the cationic lipids, and the complementary nature of those properties.
摘要翻译: 脂质颗粒可以包括多个阳离子脂质,例如第一阳离子脂质和第二阳离子脂质。 可以基于第一性质选择第一阳离子脂质,并且可以基于第二性质选择第二阳离子脂质。 第一和第二属性是互补的。 脂质颗粒的属性可以反映阳离子脂质的选择性质,以及这些性质的互补性质。
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公开(公告)号:US08450467B2
公开(公告)日:2013-05-28
申请号:US13326203
申请日:2011-12-14
CPC分类号: A61K47/28 , A61K31/70 , A61K31/7004 , A61K31/7052 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22 , A61K47/543 , A61K47/549 , A61K47/60 , A61K48/00 , C07H21/02 , Y02A50/385 , Y02A50/411 , Y02A50/467 , Y02P20/55
摘要: The present invention provides iRNA agents comprising at least one subunit of the formula (I): wherein: A and B are each independently for each occurrence O, N(RN) or S; X and Y are each independently for each occurrence H, OH, a hydroxyl protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z′)(Z″)O-nucleoside, —P(Z′)(Z″)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, —P(Z′)(Z″)O-Linker-OP(Z′″)(Z″″)O-oligonucleotide, a nucleotide, an oligonucleotide, —P(Z′)(Z″)-formula (I), —P(Z′)(Z″)— or -Linker-R; R is LG, -Linker-LG, or has the structure shown below: LG is independently for each occurrence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide; RN is independently for each occurrence H, methyl, ethyl, propyl, isopropyl, butyl, or benzyl; and Z′, Z″, Z′″ and Z″″ are each independently for each occurrence O or S.
摘要翻译: 本发明提供包含至少一个式(I)亚基的iRNA试剂:其中:A和B各自独立地为O,N(RN)或S; X和Y各自独立地为H,OH,羟基保护基,磷酸基,磷酸二酯基,活化磷酸酯基,活化亚磷酸酯基,亚磷酰胺,固体载体,-P(Z')( Z')(Z“)O - 寡核苷酸,脂质,PEG,类固醇,亲脂体,聚合物,-P(Z')(Z”)O (Z')(Z“) - (Z”)(Z“)(Z”)(Z“)(Z”) (Z“) - 或-Linker-R; R是LG,-Linker-LG,或具有如下结构:每次出现LG独立为碳水化合物,例如单糖,二糖,三糖,四糖,寡糖,多糖; 乙烯基,丙基,异丙基,丁基或苄基各自独立地为N, Z',Z“,Z”“和Z”“各自独立地为O或S.
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公开(公告)号:US20110311583A1
公开(公告)日:2011-12-22
申请号:US13128287
申请日:2009-11-10
申请人: Muthiah Manoharan , kallanthottathil G. Rajeev , Muthusamy Jayaraman , David Butler , Jayaprakash K. Narayanannair , Martin Maier , Laxman Eltepu
发明人: Muthiah Manoharan , kallanthottathil G. Rajeev , Muthusamy Jayaraman , David Butler , Jayaprakash K. Narayanannair , Martin Maier , Laxman Eltepu
IPC分类号: A61K39/12 , A61K47/08 , C07J41/00 , A61K47/28 , C07C211/22 , A61K47/16 , C07C211/21 , C07D211/72 , A61K47/22 , C07C251/78 , C07C279/12 , C07C271/12 , A61K48/00 , A61K31/713 , C12N5/00 , A61K39/39 , A61K39/00 , A61K39/02 , A61P37/04 , A61P31/12 , A61P31/04 , A61P33/00 , C07C49/203
CPC分类号: A61K47/44 , A61K9/1272 , A61K31/7088 , A61K31/713 , A61K39/00 , A61K39/39 , A61K47/10 , A61K47/18 , A61K47/20 , A61K47/28 , A61K2039/55555 , A61K2039/55561 , C07C229/08 , C07C229/30 , C07C237/16 , C07C251/38 , C07C251/78 , C07C271/12 , C07C271/20 , C07C323/25 , C07D203/10 , C07D317/28 , C07D317/44 , C07D317/46 , C07D317/72 , C07D319/06 , C07D405/12 , C07D491/056 , C07D491/113 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/3515 , C12N2320/32 , Y02A50/464
摘要: (A1) Translate this text The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure (I) wherein R1 and R2 are each independently for each occurrence optionally substituted C10-C30 alkyl, optionally substituted C10-C30 alkenyl, optionally substituted C10-C30 alkynyl, optionally substituted C10-C30 acyl, or -linker-ligand; R3 is H, optionally substituted C1-C10 alkyl, optionally substituted C2-C10 alkenyl, optionally substituted C2-C10 alkynyl, alkylhetrocycle, alkylphosphate, alkylphosphorothioate, alkylphosphorodithioate, alkylphosphonates, alkylamines, hydroxyalkyls, ?-aminoalkyls, ?-(substituted)aminoalkyls, ?-phosphoalkyls, ?-thiophosphoalkyls, optionally substituted polyethylene glycol (PEG, mw 100-40K), optionally substituted mPEG (mw 120-40K), heteroaryl, heterocycle, or linker-ligand; E is O, S, N(Q), C(O), N(Q)C(O), C(0)N(Q), (Q)N(CO)O, O(CO)N(Q), S(O), NS(O)2N(Q), S(O)2, N(Q)S(O)2, SS, O═N, aryl, heteroaryl, cyclic or heterocycle; and, Q is H, alkyl, ?-aminoalkyl, ?-(substituted)aminoalky, ?-phosphoalkyl or ?-thiophosphoalkyl.
摘要翻译: (A1)翻译本发明本发明提供有利地用于脂质颗粒以用于体内递送治疗剂至细胞的脂质。 特别地,本发明提供具有以下结构(I)的脂质,其中R1和R2各自独立地为任何取代的C 10 -C 30烷基,任选取代的C 10 -C 30烯基,任选取代的C 10 -C 30炔基,任选取代的C 10 -C 30 酰基或 - 连接子 - 配体; R 3是H,任选取代的C 1 -C 10烷基,任选取代的C 2 -C 10烯基,任选取代的C 2 -C 10炔基,烷基硫代磷酸烷基酯,硫代磷酸烷基酯,磷酸二烷基酯,烷基膦酸酯,烷基胺,羟烷基,α-氨基烷基, 任选取代的聚乙二醇(PEG,mw 100-40K),任选取代的mPEG(mw 120-40K),杂芳基,杂环或连接基 - 配体; E是O,S,N(Q),C(O),N(Q)C(O),C(O)N(Q),(Q) ),S(O),NS(O)2N(Q),S(O)2,N(Q)S(O)2,SS,O = N,芳基,杂芳基,环状或杂环; Q为H,烷基,α-氨基烷基,β-(取代)氨基烷基,α-磷酸烷基或α-硫代磷酰烷基。
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公开(公告)号:US20140200257A1
公开(公告)日:2014-07-17
申请号:US13979336
申请日:2012-01-11
申请人: Kallanthottathil G Rajeev , Muthiah Manoharan , Muthusamy Jayaraman , Martin Maier , Jayaprakash K. Nair , David Butler
发明人: Kallanthottathil G Rajeev , Muthiah Manoharan , Muthusamy Jayaraman , Martin Maier , Jayaprakash K. Nair , David Butler
IPC分类号: A61K47/18 , C07D207/12 , C07D307/20 , C07C271/16 , C07C235/06
CPC分类号: A61K47/18 , A61K47/6911 , C07C235/06 , C07C271/16 , C07D207/12 , C07D207/14 , C07D307/12 , C07D307/20 , C08G65/329 , C08L2203/02
摘要: The invention provides poly(ethylene glycol)-lipid conjugates for use in drug delivery.
摘要翻译: 本发明提供用于药物递送的聚(乙二醇)脂质缀合物。
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