公开(公告)号：US20170144967A1

公开(公告)日：2017-05-25

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：US20170101370A1

公开(公告)日：2017-04-13

申请号：US15347647

申请日：2016-11-09

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 is described is a compound of formula I wherein R is a linear alkyl of 1 to 12 carbons, or a linear alkenyl or alkynyl of 2 to 12 carbons; L is a linear alkylene or alkenylene of 5 to 18 carbons; X is —CO—O— or —O—CO—; Y is S or O; R1 is a linear or branched alkylene consisting of 1 to 6 carbons; and R2 and R3 are the same or different, consisting of a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; and n is 1-6; or a salt, a solvate, or a pharmaceutical formulation thereof.

公开(公告)号：US20170016000A1

公开(公告)日：2017-01-19

申请号：US15212279

申请日：2016-07-17

Applicant: Arcturus Therapeutics, Inc.

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

IPC: C12N15/113

CPC classification number: C12N15/1131 ,  C12N2310/14 ,  C12N2310/323 ,  C12N2310/344

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.

Abstract translation: 本发明包括可用于药物治疗方法的化合物和组合物，通常用于在受试者中抑制乙型肝炎病毒。 所述化合物具有第一链和第二链，每条链长度为19-29个单体，所述单体包含UNA单体和核酸单体，并且所述化合物靶向HBV基因组的序列。

公开(公告)号：US11939600B2

公开(公告)日：2024-03-26

申请号：US16617462

申请日：2018-05-31

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Carlos Gustavo Perez-Garcia ,  Padmanabh Chivukula ,  Hari Bhaskaran ,  Sean Christopher Daugherty ,  Christian W. Cobaugh

IPC: C12N9/02 ,  A61K31/7115 ,  A61K48/00

CPC classification number: C12N9/0071 ,  A61K31/7115 ,  A61K48/005 ,  C12Y114/16001 ,  A61K48/00

Abstract: This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human phenylalanine hydroxylase (PAH), or a fragment thereof having PAH activity. The polynucleotide and oligomer molecules are expressible to provide the human PAH or a fragment thereof having PAH activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with phenylketonuria, decreased metabolism of phenylalanine, or increased levels of phenylalanine in a subject.

公开(公告)号：US20220378702A1

公开(公告)日：2022-12-01

申请号：US17737862

申请日：2022-05-05

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kumar Rajappan ,  Steven Tanis ,  Rajesh Mukthavaram ,  Amit Sagi ,  Priya Prakash Karmali ,  Padmanabh Chivukula

IPC: A61K9/127 ,  C07K19/00 ,  A61K31/7105

Abstract: Peptides and Peptide-lipid conjugates are provided in which the peptide has the general Formula (I) wherein, A1 is selected from serine, threonine, O—C1-6 alkyl serine, and O—C1-6 alkyl threonine; A2 is selected from serine, threonine, O—C1-6 alkyl serine, and O—C1-6 alkyl threonine; A3 is selected from glutamic acid, glutamine, asparagine, and aspartic acid; A4 is proline; each A5 is independently selected from a natural or modified amino acid; The peptide-lipid conjugates can be used in lipid formulations for the delivery of nucleic acids.

公开(公告)号：US20210323914A1

公开(公告)日：2021-10-21

申请号：US17216357

申请日：2021-03-29

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula ,  Priya Karmali ,  Steven 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：US10968248B2

公开(公告)日：2021-04-06

申请号：US16600336

申请日：2019-10-11

Applicant: c/o Arcturus Therapeutics, Inc.

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

IPC: C07H21/00 ,  C07H19/16 ,  C07H19/20 ,  C07H19/207 ,  C07H21/02 ,  A61K31/7125

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：US20210002217A1

公开(公告)日：2021-01-07

申请号：US17026473

申请日：2020-09-21

Applicant: Arcturus Therapeutics, Inc.

Inventor： Joseph E. Payne ,  Padmanabh Chivukula

IPC: C07C323/52 ,  C07C271/22 ,  C07C235/12 ,  C07C237/12 ,  C12N15/11 ,  C07C323/60 ,  C07J41/00 ,  C07C333/04 ,  C11C3/04 ,  A61K47/18 ,  A61K47/20 ,  C07C323/25 ,  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：US20180135051A1

公开(公告)日：2018-05-17

申请号：US15859571

申请日：2017-12-31

Applicant: Arcturus Therapeutics, Inc.

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

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

CPC classification number: C12N15/113 ,  A61K9/127 ,  A61K31/685 ,  A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/32 ,  C12N2310/323 ,  C12N2320/32 ,  C12N2320/34

Abstract: This invention provides pharmaceutical compositions containing a UNA oligomer targeted to TTR and a pharmaceutically acceptable carrier. The compositions can be used in methods for treating or preventing TTR-related amyloidosis in a primate. The compositions, upon administering a single dose to the primate, can reduce TTR protein in the primate for a period of days to weeks.

公开(公告)号：US20180072664A1

公开(公告)日：2018-03-15

申请号：US15818424

申请日：2017-11-20

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 having the formula wherein R1 is branched alkyl of the structure (CH3(CH2)m)2CH—, wherein m is 2 or 3; R2 is a linear alkyl of 1, 2, 3, 4, 5, 6, 7, 8, or 9 carbons, a branched alkyl of 3, 4, 5, 6, 7, 8, or 9 carbons, or an alkenyl or alkynyl of 2, 3, 4, 5, 6, 7, 8, 9, 10 or 11 carbons; R3 is —(CH2)p—, wherein p is 2, 3, 4, 5 or 6; 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; L1 and L2 are the same or different, of the structure —(CH2)n—, wherein n is 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, or 18; L3 is a bond; X1 is —CO—O— whereby —L1—CO—O—R2 is formed; X2 is S or O; and X3 is —CO—O— whereby —L1—CO—O—R1 is formed; and wherein m+n+p is 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, or 27; or a pharmaceutically acceptable salt thereof.