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公开(公告)号：US20120183602A1
公开(公告)日：2012-07-19
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： A61K9/127 , A61K31/7088 , C12N5/02 , A61K47/44
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：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：US20070155686A1
公开(公告)日：2007-07-05
申请号：US11477028
申请日：2006-06-27
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3521
摘要： The features of the present invention relate to compounds, compositions and methods useful for modulating the expression of HIF-1α, such as by the mechanism of RNA interference (RNAi). The compounds and compositions include iRNA agents that can be unmodified or chemically-modified.
摘要翻译： 本发明的特征涉及用于调节HIF-1α表达的化合物，组合物和方法，例如通过RNA干扰（RNAi）机制。 化合物和组合物包括可以未修饰或化学修饰的iRNA试剂。
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公开(公告)号：US08846631B2
公开(公告)日：2014-09-30
申请号：US13521597
申请日：2011-01-14
申请人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
发明人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
CPC分类号： C12N15/113 , A61K31/713 , A61K45/06 , A61K48/00 , C12N2310/141
摘要： Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of miR-34. Also provided herein are methods for the treatment of cancer.
摘要翻译： 本文提供包含低聚化合物的组合物。 在某些实施方案中，寡聚化合物可用作miRNA模拟物。 寡聚化合物可以模拟miR-34的活性。 本文还提供了治疗癌症的方法。
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26.发明授权公开(公告)号：US08664193B2
公开(公告)日：2014-03-04
申请号：US13619657
申请日：2012-09-14
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/314 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/3521 , C12Y304/21021
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor VII gene.
摘要翻译： 本发明涉及用于抑制因子VII基因表达的双链核糖核酸（dsRNA）。
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公开(公告)号：US20130123329A1
公开(公告)日：2013-05-16
申请号：US13521597
申请日：2011-01-14
申请人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
发明人： Eric G. Marcusson , Balkrishen Bhat , Peter Linsley , Akin Akinc
IPC分类号： A61K48/00 , A61K45/06 , C12N15/113
CPC分类号： C12N15/113 , A61K31/713 , A61K45/06 , A61K48/00 , C12N2310/141
摘要： Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of miR-34. Also provided herein are methods for the treatment of cancer.
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28.发明授权公开(公告)号：US08222222B2
公开(公告)日：2012-07-17
申请号：US12554231
申请日：2009-09-04
申请人： Pamela Tan , Birgit Bramlage , Maria Frank-Kamenetsky , Kevin Fitzgerald , Akin Akinc , Victor E. Kotelianski
发明人： Pamela Tan , Birgit Bramlage , Maria Frank-Kamenetsky , Kevin Fitzgerald , Akin Akinc , Victor E. Kotelianski
CPC分类号： C12N15/1137 , A61K31/713 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition; and methods for inhibiting expression of a PCSK9 gene in a cell.
摘要翻译： 本发明涉及用于抑制PCSK9基因（PCSK9基因）表达的双链核糖核酸（dsRNA），其包含长度小于30个核苷酸的核苷酸序列的反义链，其长度通常为19-25个核苷酸 并且其与PCSK9基因的至少一部分基本上互补。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由PCSK9基因表达引起的疾病和PCSK9基因表达的方法; 以及抑制细胞中PCSK9基因表达的方法。
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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：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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