公开(公告)号：US10556861B2

公开(公告)日：2020-02-11

申请号：US15400691

申请日：2017-01-06

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  C07C271/22 ,  C07C235/12 ,  C07C237/12 ,  C07C323/60 ,  C07C323/25 ,  C07C333/04 ,  C12N15/11 ,  C12N15/113 ,  C07J41/00 ,  A61K47/20 ,  A61K47/18 ,  C11C3/04

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：US10526284B2

公开(公告)日：2020-01-07

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

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：US20180371463A1

公开(公告)日：2018-12-27

申请号：US16117994

申请日：2018-08-30

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113

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：US20180148725A1

公开(公告)日：2018-05-31

申请号：US15876127

申请日：2018-01-20

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/11

Abstract: This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of a TTR gene in a subject. The compounds have a first strand and a second strand, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of a TTR gene.

公开(公告)号：US20180105816A1

公开(公告)日：2018-04-19

申请号：US15563579

申请日：2016-04-01

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61P31/14 ,  A61P25/00 ,  A61P9/00

CPC classification number: C12N15/1131 ,  A61K31/7105 ,  A61K31/7115 ,  A61K31/7125 ,  A61K31/713 ,  A61P9/00 ,  A61P25/00 ,  A61P31/14 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/346 ,  C12N2320/51 ,  C12N2320/52 ,  A61K2300/00

Abstract: This invention provides UNA oligomers for regulating the expression of a target gene. The UNA oligomers contain UNA monomer linkers, and may contain one or more nucleotides modified with a 2′-O-methyl group, one or more nucleotides modified with a 2′-deoxy-2′-fluoro group, and one or more phosphorothioate or chiral phosphorothioate intermonomer linkages. UNA oligomers can be used as active agents for preventing or treating disease.

公开(公告)号：US20180105551A1

公开(公告)日：2018-04-19

申请号：US15788742

申请日：2017-10-19

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C07H19/16 ,  C07H19/207 ,  C07H19/20

CPC classification number: C07H19/16 ,  A61K45/06 ,  C07H19/20 ,  C07H19/207 ,  C07H21/02

Abstract: What is described is a trinucleotide cap analog comprising m7G(5′)p3-N1pN2 for increased efficiency of in vitro transcription of m7G(5′)p3-RNA, wherein m7G is N7-methylguanosine or analog, (5′)p3 is a 5′,5′-triphosphate bridge, and N1 or N2 or both ribonucleotide analogs linked to each other by a phosphate, p, and wherein the trinucleotide cap analog increases the efficiency of in vitro transcription.

公开(公告)号：US20180051262A1

公开(公告)日：2018-02-22

申请号：US15806096

申请日：2017-11-07

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N9/10 ,  C07K14/445 ,  C07K14/46 ,  C07K14/47 ,  C12N9/88 ,  C12N9/64 ,  C12N9/44 ,  C12N9/12 ,  C12N9/02 ,  C07K14/81 ,  C07K14/805 ,  C07K14/745 ,  C07K14/705 ,  C07K14/575 ,  C07K14/52 ,  C07K14/505 ,  A61K48/00 ,  A61K39/00

Abstract: This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

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.

公开(公告)号：US20170152223A1

公开(公告)日：2017-06-01

申请号：US15428766

申请日：2017-02-09

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C333/04 ,  A61K9/107 ,  A61K31/713 ,  C07J41/00 ,  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.

公开(公告)号：US09580711B2

公开(公告)日：2017-02-28

申请号：US15146783

申请日：2016-05-04

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C07J41/00 ,  C07C333/04 ,  C07C271/22 ,  C07C235/12 ,  C07C237/12 ,  A61K9/107 ,  C12N15/113 ,  C12N15/11 ,  A23D9/013 ,  A61K9/127

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。