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公开(公告)号：EP3988537A1
公开(公告)日：2022-04-27
申请号：EP21212055.4
申请日：2012-12-07
发明人： MAIER, Martin , JAYARAMAN, Muthusamy , AKINC, Akin , MATSUDA, Shigeo , KADASAMY, Pachamuthu , RAJEEV, Kallanthottathil, G. , MANOHARAN, Muthiah
IPC分类号： C07C211/09 , A61K31/713 , A61K47/18 , C07C217/08 , C07C229/12 , C07C235/06 , C07C323/12 , C07C327/22 , C07C327/28 , C07D295/08
摘要： The present invention relates to specific cationic lipids comprising a primary group and two biodegradable hydrophobic tails, as well as uses thereof in the delivery of nucleic acid molecules.
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公开(公告)号：EP3249052B1
公开(公告)日：2019-04-10
申请号：EP17162101.4
申请日：2007-05-10
发明人： TAN, Pamela , BRAMLAGE, Birgit , FRANK-KAMENETSKY, Maria , FITZGERALD, Kevin , AKINC, Akin , KOTELIANSKI, Victor E.
IPC分类号： A61K31/713 , C12N15/113
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3.发明公开
公开(公告)号：EP3249052A1
公开(公告)日：2017-11-29
申请号：EP17162101.4
申请日：2007-05-10
发明人： TAN, Pamela , BRAMLAGE, Birgit , FRANK-KAMENETSKY, Maria , FITZGERALD, Kevin , AKINC, Akin , KOTELIANSKI, Victor E.
IPC分类号： C12Q1/68 , A01N43/04 , C07H21/04 , A61K31/07 , C12N15/113
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.
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），其包含具有长度小于30个核苷酸，通常长度为19-25个核苷酸的核苷酸序列的反义链 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和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。
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4.发明公开COMPOSITIONS AND METHODS FOR INHIBITING HEPCIDIN ANTIMICROBIAL PEPTIDE (HAMP) OR HAMP-RELATED GENE EXPRESSION 审中-公开组合物和方法铁调素抗菌肽（HAMP）抑制或相关基因表达谱
公开(公告)号：EP2723861A2
公开(公告)日：2014-04-30
申请号：EP12801896.7
申请日：2012-06-21
发明人： BETTENCOURT, Brian , AKINC, Akin , SEHGAL, Alfica , FOSTER, Don , MILSTEIN, Stuart , KUCHIMANCHI, Satya , MAIER, Martin , CHARISSE, Klaus , RAJEEY, Kallanthottathil
CPC分类号： C12N15/113 , A61K31/7105 , A61K33/26 , A61K47/48092 , A61K47/549 , C12N15/1136 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/30
摘要： The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a hepcidm antimicrobial peptide (HAMP) and/or HAMP-related gene, and methods of using the dsRNA to inhibit expression of HAMP and/or HAMP-related genes.
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5.发明公开
公开(公告)号：EP2245039A2
公开(公告)日：2010-11-03
申请号：EP09739290.6
申请日：2009-01-30
IPC分类号： C07H21/04
CPC分类号： C12N15/87 , A61K48/00 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2320/35 , C12N2310/3521 , C12N2310/3533
摘要： This invention relates to optimized methods for treating diseases caused by PCSK9 gene expression.
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6.发明公开
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公开(公告)号：EP3578656A1
公开(公告)日：2019-12-11
申请号：EP19168142.8
申请日：2007-05-10
发明人： TAN, Pamela , BRAMLAGE, Birgit , FRANK-KAMENETSKY, Maria , FITZGERALD, Kevin , AKINC, Akin , KOTELIANSKI, Victor E.
IPC分类号： C12N15/113 , A61K31/713
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.
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公开(公告)号：EP2788006A1
公开(公告)日：2014-10-15
申请号：EP12809020.6
申请日：2012-12-07
发明人： MAIER, Martin , JAYARAMAN, Muthusamy , AKINC, Akin , MATSUDA, Shigeo , KADASAMY, Pachamuthu , RAJEEV, Kallanthottathil, G. , MANOHARAN, Muthiah
IPC分类号： A61K31/713 , A61K31/7088 , C07F5/02 , C07C229/12 , C07C327/22 , C07C327/32 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C251/38 , C07C327/28 , C07D317/30 , C07D235/06
CPC分类号： A61K47/18 , A61K9/1272 , A61K9/5123 , A61K31/7088 , A61K31/7105 , A61K31/713 , C07C211/09 , C07C211/10 , C07C211/11 , C07C217/08 , C07C229/12 , C07C235/06 , C07C251/38 , C07C323/12 , C07C323/58 , C07C327/22 , C07C327/28 , C07C327/32 , C07D207/32 , C07D233/54 , C07D295/08 , C07D295/12 , C07D295/14 , C07D317/30 , C07F5/022
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in 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.
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公开(公告)号：EP2416652A1
公开(公告)日：2012-02-15
申请号：EP10772775.2
申请日：2010-05-05
CPC分类号： A61K31/713 , A61K9/127 , A61K9/1277 , A61K47/6911
摘要： The invention relates to the field of delivery of nucleic acid-based agents to immune cells.
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公开(公告)号：EP2068886A2
公开(公告)日：2009-06-17
申请号：EP07853756.0
申请日：2007-10-03
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottahil G. , AKINC, Akin , JAYAPRAKASH, K. Narayanannair , JAYRAMAN, Muthusamy , MAIER, Martin A.
IPC分类号： A61K31/70
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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