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公开(公告)号:US20180245077A1
公开(公告)日:2018-08-30
申请号:US15558390
申请日:2016-03-21
发明人: Ting CHIU , Narayanan HARIHARAN , Amy C. H. LEE , Christopher Justin PASETKA , Janet Ruth PHELPS , Nicholas Michael SNEAD , Andrew Anthony WIECZOREK
IPC分类号: C12N15/113 , A61P3/06
CPC分类号: C12N15/113 , A61P3/06 , C12N15/1136 , C12N2310/14 , C12N2310/32 , C12N2310/321 , C12N2310/33 , C12N2310/3515 , C12N2320/31 , C12N2320/32 , C12N2310/3521
摘要: The present invention provides compositions comprising therapeutic nucleic acids such as siRNA that target ApoC3 and ANGPTL3 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 hypertriglyceridemia in humans).
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公开(公告)号:US09878042B2
公开(公告)日:2018-01-30
申请号:US14606905
申请日:2015-01-27
发明人: Ed Yaworski , Stephen Reid , James Heyes , Adam Judge , Ian MacLachlan
IPC分类号: C07H21/02 , C07H21/04 , A61K47/44 , A61K9/00 , A61K9/127 , A61K31/713 , C12N15/11 , C12N15/88 , C12N15/113 , A61K47/54
CPC分类号: A61K47/44 , A61K9/0019 , A61K9/1272 , A61K31/713 , A61K47/543 , C12N15/111 , C12N15/1137 , C12N15/88 , C12N2310/14 , C12N2310/321 , C12N2310/344 , C12N2320/30 , C12N2320/32 , C12N2310/3521
摘要: The present invention provides novel, serum-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 serum-stable nucleic acid-lipid particles (SNALP) comprising a nucleic acid (e.g., one or more interfering RNA molecules), methods of making the SNALP, and methods of delivering and/or administering the SNALP (e.g., for the treatment of cancer). In particular embodiments, the present invention provides tumor-directed lipid particles that preferentially target solid tumors. The tumor-directed formulations of the present invention are capable of preferentially delivering a payload such as a nucleic acid to cells of solid tumors compared to non-cancerous cells.
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公开(公告)号:US20170042814A1
公开(公告)日:2017-02-16
申请号:US15164803
申请日:2016-05-25
发明人: EDWARD YAWORSKI , KIEU LAM , LLOYD JEFFS , LORNE PALMER , IAN MacLACHLAN
IPC分类号: A61K9/127 , A61K31/713 , C12N15/113
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.
摘要翻译: 本发明提供了包含一种或多种活性剂或治疗剂的新型稳定的脂质颗粒,制备脂质颗粒的方法,以及递送和/或施用脂质颗粒的方法。 更具体地,本发明提供了包含核酸(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的方法。
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公开(公告)号:US09364435B2
公开(公告)日:2016-06-14
申请号:US14462441
申请日:2014-08-18
发明人: Edward Yaworski , Kieu Lam , Lloyd Jeffs , Lorne Palmer , Ian MacLachlan
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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公开(公告)号:US08822668B2
公开(公告)日:2014-09-02
申请号:US13928309
申请日:2013-06-26
发明人: Edward Yaworski , Kieu Lam , Lloyd Jeffs , Lorne Palmer , Ian MacLachlan
IPC分类号: C07H21/04
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.
摘要翻译: 本发明提供了包含一种或多种活性剂或治疗剂的新型稳定的脂质颗粒,制备脂质颗粒的方法,以及递送和/或施用脂质颗粒的方法。 更具体地,本发明提供了包含核酸(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的方法。
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公开(公告)号:US20140065228A1
公开(公告)日:2014-03-06
申请号:US13928309
申请日:2013-06-26
发明人: Edward Yaworski , Kieu Lam , Lloyd Jeffs , Lorne Palmer , Ian MacLachlan
IPC分类号: C12N15/113
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.
摘要翻译: 本发明提供了包含一种或多种活性剂或治疗剂的新型稳定的脂质颗粒,制备脂质颗粒的方法,以及递送和/或施用脂质颗粒的方法。 更具体地,本发明提供了包含核酸(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的方法。
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公开(公告)号:US20130172405A1
公开(公告)日:2013-07-04
申请号:US13725796
申请日:2012-12-21
发明人: Ammen P. Dhillon , Amy C.H. Lee , Ian MacLachlan
CPC分类号: C07H21/02 , A61K31/7105 , A61K31/713
摘要: The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA (e.g., dsRNA such as siRNA) that target SMAD4 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 anemia of inflammation in humans).
摘要翻译: 本发明提供了包含靶向SMAD4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,制备该脂质的方法 颗粒和递送和/或施用脂质颗粒的方法(例如,用于治疗人类的炎症性贫血)。
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公开(公告)号:US20130022649A1
公开(公告)日:2013-01-24
申请号:US13513548
申请日:2010-12-01
申请人: Ed Yaworski , James Heyes , Adam Judge , Stephen Reid , Ian MacLachlan
发明人: Ed Yaworski , James Heyes , Adam Judge , Stephen Reid , Ian MacLachlan
IPC分类号: A61K31/7088 , A61K9/14 , B82Y5/00
CPC分类号: C12N15/111 , A61K9/1271 , A61K9/1272 , A61K47/183 , C12N2320/32
摘要: The present invention provides methods of preventing, decreasing, or inhibiting the degradation of cationic lipids and/or active agents (e.g., therapeutic nucleic acids) present in lipid particles, compositions comprising lipid particles stabilized by these methods, methods of making these lipid particles, and methods of delivering and/or administering these lipid particles, e.g., for the treatment of a disease or disorder.
摘要翻译: 本发明提供了防止,降低或抑制存在于脂质颗粒中的阳离子脂质和/或活性剂(例如,治疗性核酸)的降解的方法,包含通过这些方法稳定的脂质颗粒的组合物,制备这些脂质颗粒的方法, 以及递送和/或施用这些脂质颗粒的方法,例如用于治疗疾病或病症。
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公开(公告)号:US20120328668A1
公开(公告)日:2012-12-27
申请号:US13457394
申请日:2012-04-26
申请人: Ian MacLachlan , Adam Judge
发明人: Ian MacLachlan , Adam Judge
IPC分类号: A61K31/7088 , A61K9/00 , C12N5/02 , C07H21/02
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:US20120183581A1
公开(公告)日:2012-07-19
申请号:US13253917
申请日:2011-10-05
申请人: Edward Yaworski , Kieu Lam , Lloyd Jeffs , Lorne Palmer , Ian MacLachlan
发明人: Edward Yaworski , Kieu Lam , Lloyd Jeffs , Lorne Palmer , Ian MacLachlan
IPC分类号: A61K31/713 , A61K9/00
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.
摘要翻译: 本发明提供了包含一种或多种活性剂或治疗剂的新型稳定的脂质颗粒,制备脂质颗粒的方法,以及递送和/或施用脂质颗粒的方法。 更具体地,本发明提供了包含核酸(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的方法。
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