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    METHOD OF MAKING LIPID-ENCAPSULATED RNA NANOPARTICLES
    34.
    发明申请
    METHOD OF MAKING LIPID-ENCAPSULATED RNA NANOPARTICLES 审中-公开

    公开(公告)号：US20200297634A1

    公开(公告)日：2020-09-24

    申请号：US16823212

    申请日：2020-03-18

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Priya KARMALI ,  Padmanabh Chivukula ,  Joseph E. Payne ,  Yanjie Bao

    IPC: A61K9/127 ,  A61K9/14 ,  A61K47/69 ,  A61K47/60

    Abstract: A method of producing a lipid-encapsulated RNA nanoparticle, comprising the steps a) flowing an aqueous solution comprising an RNA through a 1st tube having an inner diameter (ID) of between about 0.1″ and 0.132″; b) flowing an ethanol solution comprising lipids through a 2nd tube having an ID of between about 0.005″ and 0.02″ at one third the flow rate of the aqueous solution through the 1st tube, wherein the lipids comprise a cationic lipid; and c) mixing the ethanol solution with the aqueous solution by flowing the ethanol solution and the aqueous solution into a mixing module consisting of the 2nd tube perpendicularly joined to the 1st tube; wherein the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about 10% to 75% ethanol v/v, and wherein the lipid-encapsulated RNA nanoparticles have a bilayer structure.

    Allele selective gene editing and uses thereof
    37.
    发明授权
    Allele selective gene editing and uses thereof 有权

    公开(公告)号：US10369232B2

    公开(公告)日：2019-08-06

    申请号：US15272107

    申请日：2016-09-21

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Padmanabh Chivukula ,  Rachel Wilkie-Grantham ,  Kiyoshi Tachikawa

    IPC: A61K48/00 ,  C12N15/113 ,  C12N15/90

    Abstract: This invention encompasses compounds, structures, compositions and methods for therapeutic guide molecules that direct CRISPR gene editing. A guide molecule for directing gene editing can be allele selective, or disease allele selective, and can exhibit reduced off target activity. A guide molecule can be composed of monomers, including UNA monomers, nucleic acid monomers, and modified nucleotides, wherein the compound is targeted to a genomic DNA. The guide molecules of this invention can be used as active ingredients for editing or disrupting a gene in vitro, ex vivo, or in vivo.

    Ionizable cationic lipid for RNA delivery
    38.
    发明授权
    Ionizable cationic lipid for RNA delivery 有权

    公开(公告)号：US10227302B2

    公开(公告)日：2019-03-12

    申请号：US15893362

    申请日：2018-02-09

    Applicant: Arcturus Therapeutics, Inc.

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

    IPC: C07D211/60 ,  C07D207/16 ,  A61K31/713 ,  A61K47/54 ,  A61K9/127

    Abstract: What is described is a compound wherein R1 and R2 are the same or different, each a linear or branched alkyl consisting of 1 to 20 carbons, or a linear or branched alkenyl or alkynyl consisting of 2 to 20 carbons; L1 and L2 are the same or different, each a bond, a linear alkylene of 1-18 carbons, or a linear alkenylene consisting of 2 to 18 carbons; L3 is a bond or a linear or branched alkylene consisting of 1 to 6 carbons; L4 is a bond or a methylene; X is S or O, R3 is a linear or branched alkylene consisting of 1 to 6 carbons, and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

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