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    SYMMETRIC IONIZABLE CATIONIC LIPID FOR RNA DELIVERY
    41.
    发明申请
    SYMMETRIC IONIZABLE CATIONIC LIPID FOR RNA DELIVERY 有权
    用于RNA递送的对称离子阳离子脂质

    公开(公告)号：US20150239834A1

    公开(公告)日：2015-08-27

    申请号：US14707796

    申请日：2015-05-08

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Joseph E. Payne ,  Padmanabh Chivukula

    IPC: C07C323/25 ,  A61K47/18 ,  A61K47/20 ,  C07C333/04 ,  C07C271/22

    CPC classification number: C07C323/52 ,  A61K47/18 ,  A61K47/20 ,  C07C235/12 ,  C07C237/12 ,  C07C271/22 ,  C07C323/25 ,  C07C323/60 ,  C07C333/04 ,  C07J41/0055 ,  C11C3/04 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/32

    Abstract: What are described are compounds of formulas II, III, and IV. The compound of formula II consists of a compound in which R1 and R2 are both a linear alkyl consisting of 1 to 12 carbons, an alkenyl or alkynyl consisting of 2 to 12 carbons; L1 and L2 both consist of a linear alkylene or alkenylene consisting of 5 to 18 carbons, or forming a heterocycle with N; X is S; L3 consists of a bond or a linear alkylene consisting of 1 to 6 carbons, or forming a heterocycle with N; R3 consists of a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each consisting of hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons. The compound of formulas III and IV consists of a compound in which R1 consists of a branched alkyl with 12 to 20 carbons; R2 consists of a linear alkyl with 5 to 10 carbons or a branched alkyl with 12 to 20 carbons; L1 and L2 each consists of a bond or a linear alkyl having 1 to 3 carbon atoms; X consists of S or O; L3 consists of a bond or a lower alkyl; R3 consists of a lower alkyl; and R4 and R5 are the same or different, each consisting of a lower alkyl. The compound of formulas II, III and IV may comprise a pharmaceutically acceptable salt thereof.

    Abstract translation: 描述的是式II，III和IV的化合物。 式II化合物由其中R 1和R 2均为1至12个碳原子的直链烷基，由2至12个碳组成的烯基或炔基的化合物组成; L1和L2都由直链亚烷基或由5至18个碳组成的亚烯基组成，或与N形成杂环; X是S; L3由键或由1至6个碳组成的直链亚烷基或与N形成杂环; R3由1至6个碳原子的直链或支链亚烷基组成; 并且R 4和R 5相同或不同，各自由氢或由1至6个碳组成的直链或支链烷基组成。 式III和IV的化合物由其中R 1由具有12至20个碳的支链烷基组成的化合物组成; R 2由具有5至10个碳的直链烷基或具有12至20个碳的支链烷基组成; L1和L2各自由键或具有1至3个碳原子的直链烷基组成; X由S或O组成; L3由键或低级烷基组成; R3由低级烷基组成; 并且R 4和R 5相同或不同，各自由低级烷基组成。 式II，III和IV的化合物可以包含其药学上可接受的盐。

    IONIZABLE CATIONIC LIPID FOR RNA DELIVERY
    42.
    发明申请
    IONIZABLE CATIONIC LIPID FOR RNA DELIVERY 有权
    用于RNA交付的离子化阳离子脂质

    公开(公告)号：US20150141678A1

    公开(公告)日：2015-05-21

    申请号：US14546105

    申请日：2014-11-18

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Joseph E. Payne ,  Padmanabh Chivukula

    IPC: C11C3/04 ,  C07C333/04

    CPC classification number: C07C323/52 ,  A61K47/18 ,  A61K47/20 ,  C07C235/12 ,  C07C237/12 ,  C07C271/22 ,  C07C323/25 ,  C07C323/60 ,  C07C333/04 ,  C07J41/0055 ,  C11C3/04 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/32

    Abstract: What is described is a compound of formula (1) wherein R1 and R2 are the same or different, each a linear or branched alkyl with 1-9 carbons, or an alkenyl or alkynyl with 2 to 11 carbon atoms; L1 and L2 are the same or different, each a linear alkyl having 5 to 18 carbon atoms, or form a heterocycle with N; X1 is a bond, or is —CO—O— whereby L2-CO—O—R2 is formed; X2 is S or O; L3 is a bond or a lower alkyl, or form a heterocycle with N; R3 is a lower alkyl; and R4 and R5 are the same or different, each a lower alkyl; or a pharmaceutically acceptable salt thereof.

    Abstract translation: 描述的是式（1）的化合物，其中R 1和R 2相同或不同，各自为具有1-9个碳的直链或支链烷基或具有2至11个碳原子的烯基或炔基; L1和L2相同或不同，各自为具有5至18个碳原子的直链烷基，或与N形成杂环; X1为键，或为-CO-O-，由此形成L2-CO-O-R2; X2是S或O; L3是键或低级烷基，或与N形成杂环; R3是低级烷基; 和R 4和R 5相同或不同，各自为低级烷基; 或其药学上可接受的盐。

    LIPID NANOPARTICLES ENCAPSULATION OF LARGE RNA
    44.
    发明公开
    LIPID NANOPARTICLES ENCAPSULATION OF LARGE RNA 审中-公开

    公开(公告)号：US20240277625A1

    公开(公告)日：2024-08-22

    申请号：US18614077

    申请日：2024-03-22

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Yanjie Bao ,  Brenda Clemente ,  Priya Prakash Karmali

    IPC: A61K9/51 ,  A61K31/7105

    CPC classification number: A61K9/5192 ,  A61K31/7105

    Abstract: A method of producing lipid-encapsulated RNA nanoparticles includes flowing an aqueous solution comprising an RNA through a 1st tube having a first inner diameter (ID); the RNA comprises from about 6,000 to about 13,000 nucleotides; flowing an ethanol solution comprising lipids through a 2nd tube having a second inner diameter (ID), at a flow rate of about 0.2 to about 1 times relative to the aqueous solution through the 1st tube, the lipids comprise a cationic lipid; and mixing the ethanol solution with the aqueous solution; the first ID and second ID and flow rates through the 1st tube and 2nd tube are selected to produce a shear force sufficiently low to preserve the integrity of the RNA; the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about ethanol, the lipid-encapsulated RNA nanoparticles having a bilayer structure.

    Synthetic transfer RNA with extended anticodon loop
    50.
    发明授权
    Synthetic transfer RNA with extended anticodon loop 有权

    公开(公告)号：US11434485B2

    公开(公告)日：2022-09-06

    申请号：US16980927

    申请日：2019-03-14

    Applicant: Arcturus Therapeutics, Inc.

    Inventor： Zoya Ignatova ,  Andrew Torda ,  Marco Matthies

    IPC: C12N15/11 ,  A61K48/00

    Abstract: A synthetic transfer RNA with an extended anticodon loop. A synthetic suppressor transfer RNA useful for the treatment of a genetic disease like cystic fibrosis associated with a nonsense mutation. The synthetic transfer RNA contains an extended anticodon loop with two consecutive anticodon base triplets configured to base-pair to two consecutive codon base triplets on an mRNA. The first anticodon base triplet or the second anticodon base triplet is configured to base-pair to a stop codon base triplet on the mRNA.

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