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公开(公告)号：US20170151333A1
公开(公告)日：2017-06-01
申请号：US15261570
申请日：2016-09-09
发明人： James Heyes , Alan Martin , Mark Wood
IPC分类号： A61K47/18 , A61K47/22 , C07C229/12 , C07D249/04 , C07C217/08 , C07C237/06 , C07C327/06 , C07D233/60 , A61K31/713 , C07C271/20
CPC分类号： A61K47/18 , A61K31/713 , A61K47/14 , A61K47/183 , A61K47/186 , A61K47/22 , A61K48/0033 , C07C217/08 , C07C217/46 , C07C229/12 , C07C229/30 , C07C237/06 , C07C271/20 , C07C327/06 , C07C327/22 , C07D233/60 , C07D249/04 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2320/32
摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
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公开(公告)号：US20170007702A1
公开(公告)日：2017-01-12
申请号：US15150016
申请日：2016-05-09
发明人： James Heyes , Mark Wood , Alan Martin
IPC分类号： A61K47/18 , A61K9/14 , A61K31/7088 , A61K38/00 , C12N15/113
摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel, trialkyl, cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses.
摘要翻译： 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地，这些包括提供核酸的有效包封的新型三烷基，阳离子脂质和核酸 - 脂质颗粒，并且将体内核酸包被的核酸有效地递送到体内。 本发明的组合物是高度有效的，从而允许以相对低的剂量有效击倒特异性靶蛋白。
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公开(公告)号：US09504651B2
公开(公告)日：2016-11-29
申请号：US14304578
申请日：2014-06-13
发明人： Ian MacLachlan , Lloyd Jeffs , Lorne R. Palmer , Cory Giesbrecht
IPC分类号： A61K47/10 , A61K47/24 , B01J13/04 , A61K9/127 , A61K47/48 , A61K31/7084 , A61K31/7088 , A61K47/44
CPC分类号： A61K9/1271 , A61K9/127 , A61K9/1277 , A61K31/7084 , A61K31/7088 , A61K47/10 , A61K47/24 , A61K47/44
摘要： The present invention provides apparatus and processes for producing liposomes. By providing a buffer solution in a first reservoir, and a lipid solution in a second reservoir, continuously diluting the lipid solution with the buffer solution in a mixing chamber produces a liposome. The lipid solution preferably comprises an organic solvent, such as a lower alkanol.
摘要翻译： 本发明提供了用于生产脂质体的装置和方法。 通过在第一储存器中提供缓冲溶液，并将第二储存器中的脂质溶液连续地稀释在混合室中的缓冲溶液中的脂质溶液产生脂质体。 脂溶液优选包含有机溶剂，例如低级链烷醇。
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公开(公告)号：US09404127B2
公开(公告)日：2016-08-02
申请号：US14642452
申请日：2015-03-09
发明人： Ed Yaworski , Lloyd B. Jeffs , Lorne R. Palmer
IPC分类号： C07H21/04 , C12N15/88 , A61K9/107 , A61K9/51 , A61K31/7088 , A61K31/7105 , A61K31/712 , A61K31/713 , A61K47/14 , C12N15/113 , A61K9/127
CPC分类号： C12N15/113 , A61K9/1075 , A61K9/1272 , A61K9/1274 , A61K9/5015 , A61K9/5123 , A61K31/7088 , A61K31/7105 , A61K31/712 , A61K31/713 , A61K47/14 , A61K47/543 , C12N15/88 , C12N2310/14 , C12N2310/321 , C12N2310/3515 , C12N2320/32
摘要： The present invention provides novel, stable lipid particles having a non-lamellar structure and comprising one or more active agents or therapeutic agents, methods of making such lipid particles, and methods of delivering and/or administering such lipid particles. More particularly, the present invention provides stable nucleic acid-lipid particles (SNALP) that have a non-lamellar structure and that comprise a nucleic acid (such as one or more interfering RNA), methods of making the SNALP, and methods of delivering and/or administering the SNALP.
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公开(公告)号：US09352042B2
公开(公告)日：2016-05-31
申请号：US14380536
申请日：2013-02-22
发明人： James Heyes , Mark Wood , Alan Martin
IPC分类号： A61K47/18 , A61K9/14 , A61K31/713 , C07C229/12 , C07C217/08 , C07C271/20 , C12N15/113
CPC分类号： A61K47/18 , A61K9/145 , A61K31/7088 , A61K31/713 , A61K38/00 , C07C217/08 , C07C229/12 , C07C271/20 , C07C2601/02 , C12N15/113 , C12N2310/14 , C12N2320/32
摘要： The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel, trialkyl, cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses.
摘要翻译： 本发明提供了将治疗剂递送至细胞的组合物和方法。 特别地，这些包括提供核酸的有效包封的新型三烷基，阳离子脂质和核酸 - 脂质颗粒，并且将体内核酸包被的核酸有效地递送到体内。 本发明的组合物是高度有效的，从而允许以相对低的剂量有效击倒特异性靶蛋白。
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公开(公告)号：US20160032320A1
公开(公告)日：2016-02-04
申请号：US14642452
申请日：2015-03-09
发明人： Ed Yaworski , Lloyd B. Jeffs , Lorne R. Palmer
IPC分类号： C12N15/88 , A61K31/713 , A61K9/107 , C12N15/113
CPC分类号： C12N15/113 , A61K9/1075 , A61K9/1272 , A61K9/1274 , A61K9/5015 , A61K9/5123 , A61K31/7088 , A61K31/7105 , A61K31/712 , A61K31/713 , A61K47/14 , A61K47/543 , C12N15/88 , C12N2310/14 , C12N2310/321 , C12N2310/3515 , C12N2320/32
摘要： The present invention provides novel, stable lipid particles having a non-lamellar structure and comprising one or more active agents or therapeutic agents, methods of making such lipid particles, and methods of delivering and/or administering such lipid particles. More particularly, the present invention provides stable nucleic acid-lipid particles (SNALP) that have a non-lamellar structure and that comprise a nucleic acid (such as one or more interfering RNA), methods of making the SNALP, and methods of delivering and/or administering the SNALP.
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公开(公告)号：US08999950B2
公开(公告)日：2015-04-07
申请号：US14349619
申请日：2012-10-04
发明人： Ian MacLachlan , Amy C. H. Lee
IPC分类号： A61K48/00 , C07H21/02 , A61K9/127 , A61K31/713 , C12N15/113
CPC分类号： A61K9/127 , A61K31/713 , C07H21/02 , C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/3521 , C12Y102/01003
摘要： The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA (e.g., dsRNA such as siRNA) that target aldehyde dehydrogenase (ALDH) gene expression, lipid particles comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles (e.g., for treating alcoholism in humans).
摘要翻译： 本发明提供包含靶向醛脱氢酶（ALDH）基因表达的治疗性核酸例如干扰RNA（例如，dsRNA例如siRNA）的组合物，包含治疗性核酸的一种或多种（例如混合物）的脂质颗粒，方法 制备脂质颗粒，以及递送和/或施用脂质颗粒（例如，用于治疗人类酒精中毒）的方法。
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公开(公告)号：US20140179756A1
公开(公告)日：2014-06-26
申请号：US13952529
申请日：2013-07-26
发明人： Ian MacLachlan , Adam Judge
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K9/1272 , A61K31/713 , A61K48/00 , C12N15/111 , C12N2310/14 , C12N2310/351 , C12N2320/30 , C12N2320/51
摘要： The present invention provides chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression. Advantageously, the modified siRNA of the present invention is less immunostimulatory than its corresponding unmodified siRNA sequence and retains RNAi activity against the target sequence. The present invention also provides nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The present invention further provides methods of silencing gene expression by administering a modified siRNA to a mammalian subject. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided.
摘要翻译： 本发明提供化学修饰的siRNA分子和使用这种siRNA分子沉默靶基因表达的方法。 有利地，本发明的修饰的siRNA比其相应的未修饰的siRNA序列具有更少的免疫刺激，并保留针对靶序列的RNAi活性。 本发明还提供了包含修饰的siRNA，阳离子脂质和非阳离子脂质的核酸 - 脂质颗粒，其可进一步包含抑制颗粒聚集的共轭脂质。 本发明还提供了通过向哺乳动物受试者施用修饰的siRNA来沉默基因表达的方法。 还提供了用于鉴定和/或修饰具有免疫刺激性质的siRNA的方法。
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公开(公告)号：US20180245074A1
公开(公告)日：2018-08-30
申请号：US15570111
申请日：2016-06-06
发明人： Amy C. H. LEE , Nicholas D. WEBER
CPC分类号： C12N15/11 , A61K9/127 , A61K9/5015 , A61K38/00 , A61P31/20 , C12N9/22 , C12N15/1131 , C12N15/1137 , C12N15/907 , C12N2310/10 , C12N2310/20 , C12N2320/32 , C12N2800/80
摘要： The present invention provides compositions comprising therapeutic nucleic acids such as gRNA that target Hepatitis B virus (HBV) gene expression, lipid particles comprising one or more (e.g., a combination) of the therapeutic nucleic acids, and methods of delivering and/or administering the lipid particles (e.g., for treating HBV infection and/or HDV infection in humans).
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公开(公告)号：US20180085312A1
公开(公告)日：2018-03-29
申请号：US15670742
申请日：2017-08-07
发明人： Edward Yaworski , Kieu Lam , Lloyd Jeffs , Lorne Palmer , Ian MacLachlan
IPC分类号： A61K9/127 , C12N15/11 , C07J9/00 , A61K31/713 , A61K48/00 , C12N15/113 , C07H21/00
CPC分类号： A61K9/1272 , A61K9/1271 , A61K31/713 , A61K48/0025 , C07H21/00 , C07J9/00 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2320/32
摘要： The present invention provides novel, stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the present invention provides stable nucleic acid-lipid particles (SNALP) comprising a nucleic acid (such as one or more interfering RNA), methods of making the SNALP, and methods of delivering and/or administering the SNALP.
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