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    AROMATIC IONIZABLE CATIONIC LIPID
    132.
    发明申请
    AROMATIC IONIZABLE CATIONIC LIPID 审中-公开

    公开(公告)号：US20180050987A1

    公开(公告)日：2018-02-22

    申请号：US15799098

    申请日：2017-10-31

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Steven P. Tanis

    IPC: C07C333/04 ,  C07C271/22

    CPC classification number: C07C333/04 ,  C07C271/22

    Abstract: What is described is a compound of formula I wherein X is an alkylene or an alkenylene; Y is a monocyclic, bicyclic, or tricyclic arene or heteroarene; Z is S or O; L is a linear or branched alkylene of 1 to 6 carbons; R1 is a branched alkyl or alkenyl of 1 to 25 carbons; R2 is a alkyl or alkenyl of 1 to 20 carbons; R3 and R4 are independently a linear or branched alkyl of 1 to 6 carbons; n is 0 to 6; and m, p, q, and r are independently 1-18; or a pharmaceutically acceptable salt thereof.

    UNA SINGLE STRANDED OLIGOMERS FOR THERAPEUTICS
    137.
    发明申请
    UNA SINGLE STRANDED OLIGOMERS FOR THERAPEUTICS 审中-公开
    UNA单独使用的治疗方法

    公开(公告)号：US20160168567A1

    公开(公告)日：2016-06-16

    申请号：US15050065

    申请日：2016-02-22

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Jesper Wengel

    IPC: C12N15/113

    CPC classification number: C12N15/113 ,  C07H21/00 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2320/51

    Abstract: The present invention is directed to RNA oligonucleotides or complexes of RNA oligonucleotides, denoted herein together as RNA complexes, containing at least one, but optionally more that one, hydroxymethyl substituted nucleotide monomer(s). By a hydroxymethyl substituted nucleotide monomer is understood a nucleotide monomer containing a hydroxymethyl group (that may be unsubstituted, O-substituted for example with a conjugating group, or converted into an optionally substituted or conjugated aminomethyl group). This hydroxymethyl group is not partaking in formation of an internucleotide linkage and is not the hydroxymethyl group (containing the 5′-hydroxy group) of a natural RNA monomer. The RNA complexes of the invention may be useful for therapeutic applications, diagnostic applications or research applications. The complexes include short interfering RNA complexes (siRNA duplexes) comprising an antisense strand and a continued or a discontinued passenger strand (“sense strand”) capable of regulating gene expression. At least one of these strands, possible more than one of these strands, are modified with one or more hydroxymethyl substituted nucleotide monomer(s) of this invention, that can be positioned at the 3′-end, at the 5′-end or internally. The RNA complexes of the invention can also be single stranded RNA oligonucleotides (“RNA strands”) that are modified with at least one, but optionally more that one, hydroxymethyl substituted nucleotide monomer(s). Such single stranded RNA strands are to be considered included whenever the term RNA complexes is used in this patent application. The complexes of the invention display enhanced stability towards nucleolytic degradation relative to the corresponding complexes comprised entirely from natural RNA monomers.

    Abstract translation: 本发明涉及RNA寡核苷酸或RNA寡核苷酸的复合物，其在本文中一起表示为RNA复合物，其含有至少一个但任选地更多一个羟甲基取代的核苷酸单体。 羟甲基取代的核苷酸单体被理解为含有羟甲基（其可以是未取代的，O-取代的，例如用共轭基团或转化为任选取代或共轭的氨基甲基）的核苷酸单体。 该羟甲基不参与核苷酸间键连接的形成，不是天然RNA单体的羟甲基（含有5'-羟基）。 本发明的RNA复合物可用于治疗应用，诊断应用或研究应用。 复合物包括包含能够调节基因表达的反义链和连续或不连续的乘客链（“有义链”）的短干扰RNA复合物（siRNA双链体）。 这些链中的至少一个，可能多于一个这些链，用本发明的一个或多个羟甲基取代的核苷酸单体修饰，其可以位于5'端的3'末端，或 在内部 本发明的RNA复合物还可以是用至少一种但任选地更多一个羟甲基取代的核苷酸单体修饰的单链RNA寡核苷酸（“RNA链”）。 只要在本专利申请中使用术语RNA复合物，就认为这样的单链RNA链。 本发明的复合物相对于完全由天然RNA单体组成的相应复合物显示出增强的对于核分解降解的稳定性。

    Asymmetric ionizable cationic lipid for RNA delivery
    138.
    发明授权
    Asymmetric ionizable cationic lipid for RNA delivery 有权
    用于RNA递送的不对称可电离阳离子脂质

    公开(公告)号：US09365610B2

    公开(公告)日：2016-06-14

    申请号：US14707876

    申请日：2015-05-08

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  David Webb

    IPC: C07J41/00 ,  C07C333/04 ,  C07C271/22 ,  C07C235/12 ,  C07C237/12 ,  C12N15/11 ,  A23D9/013 ,  A61K9/127 ,  A61K9/107

    CPC classification number: C07C333/04 ,  A23D9/013 ,  A61K9/1075 ,  A61K9/1272 ,  A61K31/713 ,  C07C235/12 ,  C07C237/12 ,  C07C271/22 ,  C07J41/0055 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32

    Abstract: What is described is a compound of formula I wherein R1 consists of a linear or branched alkyl consisting of 1-18 carbons, an alkenyl or alkynyl consisting of 2 to 12 carbons, or a cholesteryl; R2 consists of a linear or branched alkyl or an alkenyl consisting of 1 to 18 carbons; L1 consists of a linear alkyl consisting of 5 to 9 carbons or, when R1 consists of a cholesteryl then L1 consists of a linear alkylene or alkenyl consisting of 3 to 4 carbons; X1 consists of —O—(CO)— or —(CO)—O—; X2 consists of S or O; L2 consists of a bond or a linear alkylene of 1 to 6 carbons; R3 consists of a linear or branched alkylene with 1 to 6 carbons; and R4 and R5 are the same or different, each consisting of a linear or branched alkyl of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

    Abstract translation: 描述的是式I的化合物，其中R 1由1-18个碳组成的直链或支链烷基，由2至12个碳组成的烯基或炔基或胆固醇基; R2由直链或支链烷基或由1至18个碳组成的烯基组成; L1由5至9个碳原子的直链烷基组成，或当R1由胆甾醇基组成时，则L1由直链亚烷基或由3至4个碳组成的烯基组成; X1由-O-（CO） - 或 - （CO）-O-组成; X2由S或O组成; L2由1至6个碳原子的键或直链亚烷基组成; R3由具有1至6个碳的直链或支链亚烷基组成; 并且R 4和R 5相同或不同，各自由1至6个碳的直链或支链烷基组成; 或其药学上可接受的盐。

    UNA OLIGOMERS HAVING REDUCED OFF-TARGET EFFECTS IN GENE SILENCING
    139.
    发明申请
    UNA OLIGOMERS HAVING REDUCED OFF-TARGET EFFECTS IN GENE SILENCING 审中-公开
    具有减少基因沉默中的非目标效应的UNA低聚体

    公开(公告)号：US20150307881A1

    公开(公告)日：2015-10-29

    申请号：US14667680

    申请日：2015-03-25

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Kiyoshi Tachikawa ,  Joseph E. Payne ,  Padmanabh Chivukula

    IPC: C12N15/113 ,  A61K47/14 ,  A61K31/713

    CPC classification number: C12N15/113 ,  A61K31/713 ,  A61K47/14 ,  C12N2310/14 ,  C12N2310/323

    Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

    Abstract translation: 本发明提供用于基因沉默的UNA寡聚体，具有降低的脱靶效应。 UNA低聚物可以具有第一链和第二链，每条链长度为19-29个单体，单体为UNA单体和各种核酸单体。 实施方案包括通过向受试者施用UNA寡聚体来治疗或预防TTR相关淀粉样变性的药物组合物和方法，其具有减少的脱靶效应。

    ASYMMETRIC IONIZABLE CATIONIC LIPID FOR RNA DELIVERY
    140.
    发明申请
    ASYMMETRIC IONIZABLE CATIONIC LIPID FOR RNA DELIVERY 有权
    用于RNA交付的不对称离子型阳离子脂质

    公开(公告)号：US20150239926A1

    公开(公告)日：2015-08-27

    申请号：US14707876

    申请日：2015-05-08

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Joseph E. Payne ,  Padmanabh Chivukula

    IPC: C07J41/00 ,  A61K47/28 ,  A61K47/20 ,  C07C333/04

    CPC classification number: C07C333/04 ,  A23D9/013 ,  A61K9/1075 ,  A61K9/1272 ,  A61K31/713 ,  C07C235/12 ,  C07C237/12 ,  C07C271/22 ,  C07J41/0055 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32

    Abstract: What is described is a compound of formula I wherein R1 consists of a linear or branched alkyl consisting of 1-18 carbons, an alkenyl or alkynyl consisting of 2 to 12 carbons, or a cholesteryl; R2 consists of a linear or branched alkyl or an alkenyl consisting of 1 to 18 carbons; L1 consists of a linear alkyl consisting of 5 to 9 carbons or, when R1 consists of a cholesteryl then L1 consists of a linear alkylene or alkenyl consisting of 3 to 4 carbons; X1 consists of —O—(CO)— or —(CO)—O—; X2 consists of S or O; L2 consists of a bond or a linear alkylene of 1 to 6 carbons; R3 consists of a linear or branched alkylene with 1 to 6 carbons; and R4 and R5 are the same or different, each consisting of a linear or branched alkyl of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

    Abstract translation: 描述的是式I的化合物，其中R 1由1-18个碳组成的直链或支链烷基，由2至12个碳组成的烯基或炔基或胆固醇基; R2由直链或支链烷基或由1至18个碳组成的烯基组成; L1由5至9个碳原子的直链烷基组成，或当R1由胆甾醇基组成时，则L1由直链亚烷基或由3至4个碳组成的烯基组成; X1由-O-（CO） - 或 - （CO）-O-组成; X2由S或O组成; L2由1至6个碳原子的键或直链亚烷基组成; R3由具有1至6个碳的直链或支链亚烷基组成; 并且R 4和R 5相同或不同，各自由1至6个碳的直链或支链烷基组成; 或其药学上可接受的盐。

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