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。

公开(公告)号：US20160130567A1

公开(公告)日：2016-05-12

申请号：US14929366

申请日：2015-11-01

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N9/10 ,  C07K14/505 ,  C07K14/81 ,  C07K14/705 ,  C12N9/88 ,  C12N9/24 ,  C07K14/575 ,  C07K14/47 ,  C12N9/12 ,  C07K14/445 ,  C07K14/005 ,  C12N9/64 ,  C12N9/02

CPC classification number: C12N9/1018 ,  A61K39/00 ,  A61K48/00 ,  C07K14/445 ,  C07K14/463 ,  C07K14/47 ,  C07K14/4712 ,  C07K14/4717 ,  C07K14/505 ,  C07K14/524 ,  C07K14/575 ,  C07K14/705 ,  C07K14/745 ,  C07K14/805 ,  C07K14/8125 ,  C12N9/0071 ,  C12N9/1051 ,  C12N9/1205 ,  C12N9/2451 ,  C12N9/644 ,  C12N9/88 ,  C12N2740/16222 ,  C12N2840/105 ,  C12N2840/60 ,  C12Y114/16001 ,  C12Y201/03003 ,  C12Y204/01025 ,  C12Y207/0104 ,  C12Y302/01033 ,  C12Y304/21022 ,  C12Y403/02001

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.

Abstract translation: 本发明提供一系列可翻译的信使UNA（mUNA）分子。 可以在体外和体内翻译mUNA分子以提供活性多肽或蛋白质，或提供免疫剂或疫苗组分。 mUNA分子可用作活性剂以在细胞或受试者中表达活性多肽或蛋白质。 其中，mUNA分子可用于治疗罕见疾病的方法。

公开(公告)号：US09303260B2

公开(公告)日：2016-04-05

申请号：US14702991

申请日：2015-05-04

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: This invention provides UNA oligomers for therapeutics that can be used to modulate or reduce gene expression. The UNA oligomers can be composed of one or more 2′-3′-seco-nucleomonomers and one or more natural or non-natural nucleotide monomers, and can be a duplex having first and second strands. Embodiments include UNA oligomers with phosphorothioate or boranophosphate intermonomer linkages. The UNA oligomers can provide reduced off target effects while modulating gene expression.

Abstract translation: 本发明提供了可用于调节或降低基因表达的治疗剂的UNA寡聚体。 UNA低聚物可以由一个或多个2'-3'-中间核单体和一种或多种天然或非天然核苷酸单体组成，并且可以是具有第一和第二链的双链体。 实施方案包括具有硫代磷酸酯或硼酸甘油酯单体间连接的UNA低聚物。 UNA寡聚体可以在调节基因表达的同时提供减少的靶效应。

公开(公告)号：US20150232849A1

公开(公告)日：2015-08-20

申请号：US14702991

申请日：2015-05-04

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: This invention provides UNA oligomers for therapeutics that can be used to modulate or reduce gene expression. The UNA oligomers can be composed of one or more 2′-3′-seco-nucleomonomers and one or more natural or non-natural nucleotide monomers, and can be a duplex having first and second strands. Embodiments include UNA oligomers with phosphorothioate or boranophosphate intermonomer linkages. The UNA oligomers can provide reduced off target effects while modulating gene expression.

Abstract translation: 本发明提供了可用于调节或降低基因表达的治疗剂的UNA寡聚体。 UNA低聚物可以由一个或多个2'-3'-中间核单体和一种或多种天然或非天然核苷酸单体组成，并且可以是具有第一和第二链的双链体。 实施方案包括具有硫代磷酸酯或硼酸甘油酯单体间连接的UNA低聚物。 UNA寡聚体可以在调节基因表达的同时提供减少的靶效应。

公开(公告)号：US12290567B2

公开(公告)日：2025-05-06

申请号：US17011880

申请日：2020-09-03

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar Rajappan ,  Padmanabh Chivukula ,  Kiyoshi Tachikawa ,  Steven Tanis ,  Priya Karmali

IPC: A61K47/54 ,  C07H15/04 ,  C12N15/113

Abstract: ASGP-R binding molecular conjugates are provided. The conjugates are useful to deliver therapeutically effective amounts of biologically active molecules to target cells and tissues of a subject. Compositions are also provided comprising the molecular conjugates.

公开(公告)号：US12286395B2

公开(公告)日：2025-04-29

申请号：US17026473

申请日：2020-09-21

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  A61K47/18 ,  A61K47/20 ,  C07C235/12 ,  C07C237/12 ,  C07C271/22 ,  C07C323/25 ,  C07C323/60 ,  C07C333/04 ,  C07J41/00 ,  C11C3/04 ,  C12N15/11 ,  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：US20250049950A1

公开(公告)日：2025-02-13

申请号：US18732954

申请日：2024-06-04

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Carlos G. PEREZ-GARCIA ,  Kiyoshi TACHIKAWA ,  Daiki MATSUDA ,  Padmanabh CHIVUKULA ,  Priya Prakash KARMALI ,  Yanjie BAO ,  Jerel Boyd Lee VEGA ,  Rajesh MUKTHAVARAM ,  Amit SAGI

IPC: A61K48/00 ,  A61P11/00 ,  C07K14/47

Abstract: Nucleotides encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein are provided herein. Also describe are mRNA constructs that can be used to express CFTR protein in vitro or in vivo. The mRNA constructs can be formulated in a lipid formulation and administered via inhalation to treat cystic fibrosis.

公开(公告)号：US12178921B2

公开(公告)日：2024-12-31

申请号：US17402077

申请日：2021-08-13

Applicant: Arcturus Therapeutics, Inc.

Inventor： Amit Sagi ,  Yanjie Bao ,  Priya Prakash Karmali

IPC: A61K9/51 ,  A61K9/19 ,  A61K48/00

Abstract: Methods of preparing lyophilized lipid nanoparticle-nucleic acid compositions are provided. The methods comprise preparing a suspension of lipid nanoparticles with a monosaccharide and one or more excipients selected from thiosulfate, potassium sorbate, sodium benzoate, and iodixanol. Lyophilized lipid nanoparticle-nucleic acid compositions and methods of reconstituting and administering the same are further provided.

公开(公告)号：US20240301007A1

公开(公告)日：2024-09-12

申请号：US18427625

申请日：2024-01-30

Applicant: Arcturus Therapeutics, Inc.

Inventor： Brian SULLIVAN ,  Sean SULLIVAN ,  Daiki MATSUDA ,  Rodrigo YELIN ,  Jinho PARK

IPC: C07K14/005 ,  A61K9/127 ,  A61P31/16

CPC classification number: C07K14/005 ,  A61K9/1272 ,  A61P31/16 ,  C12N2760/16121 ,  C12N2760/16134 ,  C12N2770/36122 ,  C12N2770/36134

Abstract: Provided herein are RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof. Also provided herein are compositions that include RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof, and lipids. RNA molecules and compositions including them are useful for inducing immune responses.