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公开(公告)号：WO2018006052A1
公开(公告)日：2018-01-04
申请号：PCT/US2017/040446
申请日：2017-06-30
申请人： PROTIVA BIOTHERAPEUTICS, INC. , ABRAMS, Michael J. , HEYES, James , JUDGE, Adam , LAM, Kieu Mong , PALMER, Lorne Ralph , REID, Stephen P. , YAWORSKI, Edward D.
发明人： ABRAMS, Michael J. , HEYES, James , JUDGE, Adam , LAM, Kieu Mong , PALMER, Lorne Ralph , REID, Stephen P. , YAWORSKI, Edward D.
摘要： The present invention provides compositions comprising nucleic acid molecules, such as mRNA molecules, encapsulated within lipid particles. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease.
摘要翻译： 本发明提供包含封装在脂质颗粒内的核酸分子例如mRNA分子的组合物。 例如，所述组合物可用于将mRNA分子引入人受试者中，在所述人受试者中它们被翻译以产生用于改善疾病的一种或多种症状的多肽。
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公开(公告)号：WO2017120527A2
公开(公告)日：2017-07-13
申请号：PCT/US2017/012614
申请日：2017-01-06
申请人： PROTIVA BIOTHERAPEUTICS, INC. , CUCONATI, Andrea , LEE, Amy C. H. , RIJNBRAND, Cornelis A. , SOFIA, Michael J.
IPC分类号： A61K48/00
CPC分类号： A61K31/713 , A61K31/18 , A61K31/352 , A61K31/4375 , A61K31/5377 , A61K31/675 , A61K45/06 , A61K2300/00
摘要： The invention provides therapeutic combinations and therapeutic methods that are useful for treating Hepatitis B.
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3.发明申请COMPOSITIONS AND METHODS FOR SILENCING HEPATITIS B VIRUS GENE EXPRESSION 审中-公开用于沉默乙型肝炎病毒基因表达的组合物和方法
公开(公告)号：WO2017019891A2
公开(公告)日：2017-02-02
申请号：PCT/US2016/044541
申请日：2016-07-28
IPC分类号： C12N15/113
CPC分类号： C12N15/1131 , C12N2310/14 , C12N2310/32 , C12N2320/31 , C12N2320/32 , C12N2310/321 , C12N2310/3521
摘要： The present invention provides compositions comprising therapeutic nucleic acids such as siRNA 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).
摘要翻译： 本发明提供包含靶向乙型肝炎病毒（HBV）基因表达的治疗性核酸如siRNA的组合物，包含治疗性核酸的一种或多种（例如，组合）的脂质颗粒，以及递送和/或施用 脂质颗粒（例如，用于治疗人类的HBV感染和/或HDV感染）。
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4.发明申请COMPOSITIONS AND METHODS FOR SILENCING EXPRESSION OF HEPATITIS D VIRUS RNA 审中-公开用于沉默肝炎病毒RNA表达的组合物和方法
公开(公告)号：WO2016183366A3
公开(公告)日：2016-12-29
申请号：PCT/US2016032188
申请日：2016-05-12
申请人： PROTIVA BIOTHERAPEUTICS INC , LEE AMY C H , MACLACHLAN IAN , SNEAD NICHOLAS MICHAEL , THI EMILY P , YE XIN
发明人： LEE AMY C H , MACLACHLAN IAN , SNEAD NICHOLAS MICHAEL , THI EMILY P , YE XIN
CPC分类号： C07H21/02 , C12N15/1131 , C12N2310/11 , C12N2310/14 , C12N2310/323 , C12N2310/321 , C12N2310/3521
摘要： The present invention provides compositions comprising therapeutic nucleic acids such as siRNA that target Hepatitis D virus (HDV) RNA 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 HDV infection in humans).
摘要翻译： 本发明提供了包含治疗性核酸的组合物，所述治疗性核酸例如靶向肝炎D病毒（HDV）RNA表达的siRNA，包含一种或多种（例如，组合）治疗性核酸的脂质颗粒，以及递送和/或施用 脂质颗粒（例如，用于治疗人类中的HDV感染）。
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公开(公告)号：WO2013126803A1
公开(公告)日：2013-08-29
申请号：PCT/US2013/027469
申请日：2013-02-22
发明人： HEYES, James , WOOD, Mark , MARTIN, Alan
IPC分类号： C07C271/20 , C07C217/08 , C07C229/12
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：WO2012000104A1
公开(公告)日：2012-01-05
申请号：PCT/CA2011/000778
申请日：2011-06-30
IPC分类号： A61K31/713 , A61K31/7088 , A61K47/24 , A61K47/28 , A61K9/14
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.
摘要翻译： 本发明提供了具有非层状结构并且包含一种或多种活性剂或治疗剂的新颖稳定的脂质颗粒，制备这种脂质颗粒的方法，以及递送和/或施用这种脂质颗粒的方法。 更具体地，本发明提供了具有非层状结构并且包含核酸（例如一种或多种干扰RNA）的稳定核酸 - 脂质颗粒（SNALP），制备SNALP的方法，以及递送和 /或施用SNALP。
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公开(公告)号：WO2011141704A1
公开(公告)日：2011-11-17
申请号：PCT/GB2011/000722
申请日：2011-05-12
发明人： HEYES, James , WOOD, Mark , MARTIN, Alan
IPC分类号： C07C217/28 , C07C229/08 , C07D317/28 , A61K48/00 , A61K47/48 , C12N15/11 , C12N15/88 , C07C229/12
CPC分类号： C07C217/28 , A61K9/1272 , A61K9/5123 , A61K31/7105 , A61K31/713 , A61K47/18 , C07C229/12 , C07C2601/02 , C07D317/28 , C12N15/111 , C12N2310/14 , C12N2310/3515 , 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.
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，这些包括新型阳离子脂质和核酸 - 脂质颗粒，其提供核酸的有效包封以及将体内核酸包封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。 此外，与本领域已知的组合物和方法相比，本发明的组合物和方法毒性低，并提供更大的治疗指数。
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公开(公告)号：WO2011141703A1
公开(公告)日：2011-11-17
申请号：PCT/GB2011/000721
申请日：2011-05-12
申请人： PROTIVA BIOTHERAPEUTICS INC. , HEYES, James , WOOD, Mark , MARTIN, Alan , LEE, Amy, C., H. , JUDGE, Adam , ROBBINS, Marjorie , MACLACHLAN, Ian
发明人： HEYES, James , WOOD, Mark , MARTIN, Alan , LEE, Amy, C., H. , JUDGE, Adam , ROBBINS, Marjorie , MACLACHLAN, Ian
IPC分类号： C12N15/113 , C12N15/88 , A61K31/713 , C07C229/08 , C07D317/28
CPC分类号： A61K31/713 , C12N15/111 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/32
摘要： The present invention provides compositions and methods for the delivery of interfering RNAs such as siRNAs that silence APOB expression in cells such as liver cells. In particular, the nucleic acid-lipid particles provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells such as liver cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of APOB 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.
摘要翻译： 本发明提供了用于递送干扰RNA的组合物和方法，例如使细胞如肝细胞中的APOB表达沉默的siRNA。 特别地，核酸 - 脂质颗粒提供了核酸的有效包封以及将体内核酸封装到细胞例如肝细胞的有效递送。 本发明的组合物是非常有效的，从而允许在相对低的剂量下有效击倒APOB。 此外，与本领域先前已知的组合物和方法相比，本发明的组合物和方法毒性低，并提供更大的治疗指数。
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公开(公告)号：WO2011038160A2
公开(公告)日：2011-03-31
申请号：PCT/US2010/050077
申请日：2010-09-23
申请人： PROTIVA BIOTHERAPEUTICS, INC. , THE GOVERNMENT OF THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES , JUDGE, Adam , LEE, Yun-Han , MACLACHLAN, Ian , THORGEIRSSON, Snorri, S.
IPC分类号： C12N15/113 , A61K31/713 , A61P35/00 , C07H21/02
CPC分类号： C12N15/113 , A61K31/7105 , A61K31/713 , C12N15/1137 , C12N2310/14 , C12N2310/344 , C12N2320/31 , C12N2320/32
摘要： The present invention provides therapeutic nucleic acids such as interfering RNA (e.g., siRNA) that target the expression of genes associated with tumorigenesis and/or cell transformation, lipid particles (e.g., nucleic acid-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 the treatment of a cell proliferative disorder such as cancer.
摘要翻译： 本发明提供了治疗性核酸，例如靶向与肿瘤发生和/或细胞转化相关的基因的表达的干扰RNA（例如，siRNA），脂质颗粒（例如，核酸 - 脂质颗粒） 包含治疗性核酸的一种或多种（例如混合物），制备脂质颗粒的方法以及递送和/或施用脂质颗粒的方法，例如用于治疗细胞增殖性病症如癌症。 / p>
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10.发明申请
公开(公告)号：WO2007048046A9
公开(公告)日：2007-06-14
申请号：PCT/US2006041336
申请日：2006-10-20
申请人： PROTIVA BIOTHERAPEUTICS INC , MACLACHLAN IAN , SOOD VANDANA , GEISBERT THOMAS W , HENSLEY LISA E , KAGAN ELLIOTT
IPC分类号： A61K9/127 , A61K31/713 , C12N15/113 , C12N15/88
CPC分类号： C12N15/1131 , C12N2310/14
摘要： The present invention provides siRNA molecules that target Filovirus gene expression and methods of using such siRNA molecules to silence Filovirus gene expression. The present invention also provides nucleic acid-lipid particles that target Filovirus gene expression comprising an siRNA that silences Filovirus gene expression, a cationic lipid, and a non-cationic lipid.
摘要翻译： 本发明提供了靶向丝裂病毒基因表达的siRNA分子以及使用这种siRNA分子沉默丝状病毒基因表达的方法。 本发明还提供靶向包含沉默丝状病毒基因表达的siRNA，阳离子脂质和非阳离子脂质的siRNA的核酸 - 脂质颗粒。
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