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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：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：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：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：US07928217B2
公开(公告)日：2011-04-19
申请号：US11139089
申请日：2005-05-27
申请人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Stage Zimmermann , Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc
发明人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Stage Zimmermann , Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
摘要翻译： 本发明涉及在细胞和生物流体中具有改善的稳定性的修饰的双链寡核糖核酸（dsRNA），以及制备和鉴定具有改善的稳定性的dsRNA的方法，以及使用dsRNA抑制靶基因的表达或功能。
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公开(公告)号：US08802644B2
公开(公告)日：2014-08-12
申请号：US13357856
申请日：2012-01-25
申请人： Jianxin Chen , Steven Ansell , Akin Akinc , Joseph Robert Dorkin , Xiaojun Qin , William Cantley , Muthiah Manoharan , Kallanthottathil G. Rajeev , Jayaprakash K. Narayanannair , Muthusamy Jayaraman
发明人： Jianxin Chen , Steven Ansell , Akin Akinc , Joseph Robert Dorkin , Xiaojun Qin , William Cantley , Muthiah Manoharan , Kallanthottathil G. Rajeev , Jayaprakash K. Narayanannair , Muthusamy Jayaraman
IPC分类号： C12N15/113 , C07C229/06 , A61K47/12 , A61K47/14
CPC分类号： A61K9/1272 , A61K9/1278 , A61K31/22 , A61K31/7088 , A61K31/712 , A61K31/713 , A61K38/1709 , A61K38/4846 , A61K47/14 , A61K47/549 , A61K47/551 , A61K47/6911 , A61K48/0033 , C07C29/58 , C07C227/18 , C07C227/40 , C07C229/12 , C07D475/04 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , Y02A50/463 , A61K2300/00 , C12N2310/3521 , C12N2310/3533
摘要： The invention features a cationic lipid of formula I, an improved lipid formulation comprising a cationic lipid of formula I and corresponding methods of use. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
摘要翻译： 本发明的特征在于式I的阳离子脂质，包含式I的阳离子脂质和相应的使用方法的改进的脂质制剂。 还公开了靶向脂质和包含这种靶向脂质的特定脂质制剂。
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公开(公告)号：US08334373B2
公开(公告)日：2012-12-18
申请号：US13038672
申请日：2011-03-02
申请人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Stage Zimmermann , Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc
发明人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Stage Zimmermann , Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
摘要翻译： 本发明涉及在细胞和生物流体中具有改善的稳定性的修饰的双链寡核糖核酸（dsRNA），以及制备和鉴定具有改善的稳定性的dsRNA的方法，以及使用dsRNA抑制靶基因的表达或功能。
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