公开(公告)号：US20180327471A1

公开(公告)日：2018-11-15

申请号：US15907123

申请日：2018-02-27

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Padmanabh Chivukula ,  Daiki Matsuda ,  Arisa Cale

IPC: C07K14/505 ,  C07K14/745 ,  C07K14/81 ,  C07K14/575

CPC classification number: C07K14/505 ,  A61K31/7088 ,  A61K31/7105 ,  A61K35/545 ,  A61K38/00 ,  A61K38/17 ,  C07K14/575 ,  C07K14/745 ,  C07K14/81

Abstract: A range of therapeutic mRNA molecules expressible to provide a target polypeptide or protein. The RNA molecules can contain one or more 5-methoxyuridines and 5-methylcytidines. Further provided are DNA templates, which can be transcribed to provide a target mRNA, and can have altered nucleotides, such as reduced deoxyadenosines. This invention also provides processes for making the therapeutic mRNA molecules. The RNA molecules can be translated in vitro or in vivo to provide an active polypeptide or protein.

公开(公告)号：US20180208555A1

公开(公告)日：2018-07-26

申请号：US15925670

申请日：2018-03-19

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  C12N15/113

Abstract: What is described is a method of synthesis of the compound of formula 1A, or a salt thereof, wherein R3 is a linear or branched alkene of 1, 2, 3, 4, 5 or 6 carbons; R4 and R5 are the same or different, each a hydrogen, or a linear or branched alkyl of 1, 2, 3, 4, 5 or 6 carbons; and L3 is a bond or an alkane of 1, 2, 3, 4, 5 or 6 carbons.

公开(公告)号：US20180170866A1

公开(公告)日：2018-06-21

申请号：US15849573

申请日：2017-12-20

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Priya Karmali ,  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 consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons, or a branched chain alkyl consisting of 10 to 31 carbons; L1 and L2 are the same or different, each a linear alkane of 1 to 20 carbons or a linear alkene of 2 to 20 carbons; X1 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.

公开(公告)号：US20180170865A1

公开(公告)日：2018-06-21

申请号：US15899314

申请日：2018-02-19

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/25 ,  C07C333/04 ,  A61K47/18 ,  A61K47/20 ,  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 is described is a compound of formula I wherein R1 is a linear alkyl, alkenyl, or alkynyl of 2 to 12 carbons, or a branched alkyl with 25, 24, 23, 22, 21, 20, 19, 18, 17, 16, 15, 14, 13, 12, 11, 10, 9, 8, or 7 carbons, R2 is a branched alkyl with 25, 24, 23, 22, 21, 20, 19, 18, 17, 16, 15, 14, 13, 12, 11, 10, 9, 8, or 7 carbons, L1 and L2 are independently a linear alkylene with 1-18 carbons or alkenylene with 2-18 carbons, X is S or O, L3 is a bond or an alkylene with 1 to 6 carbons, R3 is an alkylene with 1 to 6 carbons, and R4 and R5 are the same or different, each a linear or branched alkyl with 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

公开(公告)号：US09896413B2

公开(公告)日：2018-02-20

申请号：US15614499

申请日：2017-06-05

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/25 ,  A61K47/18 ,  A61K47/20 ,  C07C271/22 ,  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 I wherein R1 is a linear alkyl, alkenyl, or alkynyl of 2 to 12 carbons, or a branched alkyl with 12 to 20 carbons, R2 is a branched alkyl with 12 to 20 carbons, L1 and L2 are independently a linear alkylene with 1-18 carbons or alkenylene with 2-18 carbons, X is S or O, L3 is a bond or an alkylene with 1 to 6 carbons, R3 is an alkylene with 1 to 6 carbons, and R4 and R5 are the same or different, each a linear or branched alkyl with 1 to 6 carbons.

公开(公告)号：US09850202B2

公开(公告)日：2017-12-26

申请号：US15423008

申请日：2017-02-02

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  C12N15/113

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 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 carbons; L1 and L2 are the same or different, each a linear alkyl having 3 to 18 carbons; X1 and X3 are —CO—O—; X3 is S or O; L3 is a lower alkyl; R3 is a lower alkyl; and R4 and R5 are the same or different, each a lower alkyl; or a pharmaceutically acceptable salt thereof.

公开(公告)号：US20170137821A1

公开(公告)日：2017-05-18

申请号：US15410984

申请日：2017-01-20

Applicant: Arcturus Therapeutics, Inc.

Inventor： Pattraranee Limphong ,  Kiyoshi Tachikawa ,  Christine Esau ,  Padmanabh Chivukula

IPC: C12N15/113 ,  A61K9/127

CPC classification number: C12N15/1131 ,  A61K9/1271 ,  A61K47/6911 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/344 ,  C12N2310/3515 ,  C12N2320/32

Abstract: This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting Hepatitis B virus in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of an HBV genome.

公开(公告)号：US20170114010A1

公开(公告)日：2017-04-27

申请号：US15400691

申请日：2017-01-06

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C333/04 ,  C07C235/12 ,  C12N15/113 ,  A61K47/20 ,  A61K47/18 ,  C07C323/25 ,  C07C271/22

Abstract: What is described is a compound of formula III or IV wherein R1 is a branched alkyl with 12 to 20 carbons; R2 is a linear alkyl with 5 to 10 carbons or a branched alkyl with 12 to 20 carbons; L1 and L2 are each a bond or a linear alkylene having 1 to 3 carbon atoms, when L3 is a bond; L1 and L2 are each a linear alkylene or alkenylene 5 to 18 carbon atoms, when L3 is an alkylene of 5 to 18 carbons; X is S or O; R3 is of a lower alkyl; and R4 and R5 are the same or different, each a lower alkyl; or a pharmaceutically acceptable salt thereof.

公开(公告)号：US09593077B2

公开(公告)日：2017-03-14

申请号：US14546105

申请日：2014-11-18

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07J41/00 ,  C07C323/25 ,  C07C271/22 ,  C07C333/04 ,  C07C235/12 ,  C07C237/12 ,  A61K47/20 ,  C12N15/11 ,  C11C3/04 ,  A61K47/18 ,  C07C323/60

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.

公开(公告)号：US20160244761A1

公开(公告)日：2016-08-25

申请号：US15146783

申请日：2016-05-04

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113

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 lipid particle comprising the compound of formula I wherein R1 consists of alkyl, alkenyl, alkynyl, or cholesteryl; R2 consists of alkyl or alkenyl; L1 consists of alkyl, alkylene or alkenyl; X1 consists of —O—(CO)— or —(CO)—O—; X2 consists of S or O; L2 consists of a bond or alkylene; R3 consists of alkylene; and R4 and R5 are the same or different, each consisting of alkyl; or a pharmaceutical preparation thereof.

Abstract translation: 描述的是包含式I化合物的脂质颗粒，其中R 1由烷基，烯基，炔基或胆固醇基组成; R2由烷基或烯基组成; L1由烷基，亚烷基或烯基组成; X1由-O-（CO） - 或 - （CO）-O-组成; X2由S或O组成; L2由键或亚烷基组成; R3由亚烷基组成; 并且R 4和R 5相同或不同，各自由烷基组成; 或其药物制剂。