公开(公告)号：US20230059111A1

公开(公告)日：2023-02-23

申请号：US17812576

申请日：2022-07-14

Applicant: ARCTURUS THERAPEUTICS, INC.

Inventor： Pattraranee LIMPHONG ,  Kiyoshi TACHIKAWA ,  Padmanabh CHIVUKULA ,  Daiki MATSUDA ,  Arisa CALE

IPC: C07K14/505 ,  C07K14/745 ,  C07K14/575 ,  C07K14/81 ,  A61K38/48 ,  A61K38/17 ,  A61K38/18 ,  A61K31/7115

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. Also provided are 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. The RNA molecules can be included in a composition used for preventing, treating, or ameliorating at least one symptom of a disease or condition in a subject in need thereof.

公开(公告)号：US11407800B2

公开(公告)日：2022-08-09

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: C12N15/10 ,  C07K14/505 ,  C07K14/745 ,  C07K14/575 ,  C07K14/81 ,  A61K38/48 ,  A61K38/17 ,  A61K38/18 ,  A61K31/7115 ,  A61K38/00

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. Also provided are 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. The RNA molecules can be included in a composition used for preventing, treating, or ameliorating at least one symptom of a disease or condition in a subject in need thereof.

公开(公告)号：US20220023442A1

公开(公告)日：2022-01-27

申请号：US17246558

申请日：2021-04-30

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 ,  C07K14/47 ,  A61P11/00

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：US20210252163A1

公开(公告)日：2021-08-19

申请号：US17234571

申请日：2021-04-19

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Priya Prakash Karmali ,  Steven Tanis

IPC: A61K48/00 ,  C07C333/04 ,  A61K9/51 ,  A61K47/20 ,  A61P31/00

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; L1 and L2 are the same or different, each a linear alkylene or alkenylene consisting 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：US10961188B2

公开(公告)日：2021-03-30

申请号：US16709800

申请日：2019-12-10

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C07C333/04 ,  C07C271/22

Abstract: Disclosed herein is a compound of Formula I, wherein 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 or solvate thereof.

公开(公告)号：US10781169B2

公开(公告)日：2020-09-22

申请号：US15925670

申请日：2018-03-19

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

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

Abstract: Disclosed herein is a lipid composition comprising a compound having the formula IA, or a pharmaceutically acceptable salt thereof, wherein R3 is a linear or branched alkylene of 1-6 carbons; R4 and R5 are the same or different, each a hydrogen, or a linear or branched alkyl of 1-6 carbons; and L3 is a bond or an alkane of 1-6 carbons.

公开(公告)号：US10731154B2

公开(公告)日：2020-08-04

申请号：US15550884

申请日：2016-02-15

Applicant: Arcturus Therapeutics, Inc.

Inventor： Jesper Wengel

IPC: C07H21/04 ,  C12N15/113 ,  C12N15/115 ,  C12N15/11 ,  C12Q1/6876

Abstract: A single stranded oligonucleotide containing two or more acyl-amino-LNA or hydrocarbyl-amino-LNA nucleotide monomers, in which other nucleotide monomers can be DNA, RNA or chemically modified nucleotide monomers; in which the monomers of the oligonucleotide are linked by phosphodiester linkages and/or phosophorothioate linkages and/or phosphotriester linkages, in which the acyl and hydrocarbyl groups of the acyl-amino-LNA and hydrocarbyl-amino-LNA monomers are optionally substituted and thus optionally contain one or more hydroxyl group(s), amino group(s), thio group(s), oxo group(s), alkylthio group(s), ether group(s), and/or thiol (mercapto) group(s), and in which the acyl and hydrocarbyl groups of the acyl-amino-LNA and hydrocarbyl-amino-LNA monomers are linear or branched chains, cyclic or a combination of both, provided that the total number of carbon atoms in each acyl and hydrocarbyl group is less than 30.

公开(公告)号：US10683500B2

公开(公告)日：2020-06-16

申请号：US16109231

申请日：2018-08-22

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113 ,  A61K31/713 ,  A61K47/14

Abstract: This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

公开(公告)号：US10399937B2

公开(公告)日：2019-09-03

申请号：US15899314

申请日：2018-02-19

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

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

Abstract: What is described herein are cationic lipids that facilitate the intracellular delivery of biologically active molecules, comprising a compound of formula I wherein R1 is a linear alkyl, alkenyl, or alkynyl of 2 to 12 carbons, or a branched alkyl with 7-25 R2 is a branched alkenyl with 7-25 carbons, L1 and L2 are independently a linear alkenylene with 2-18 carbons, X is S or O, L3 is a bond, and R3 R4, and R5 are each a linear or branched alkylene with 1-6 carbons.

公开(公告)号：US10383952B2

公开(公告)日：2019-08-20

申请号：US15387067

申请日：2016-12-21

Applicant: Arcturus Therapeutics, Inc.

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

IPC: A61K47/20 ,  A61K48/00 ,  C07C333/04 ,  A61K9/51

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; L1 and L2 are the same or different, each a linear alkylene of 1 to 20 carbons or a linear alkenylene 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.