公开(公告)号：US09962449B2

公开(公告)日：2018-05-08

申请号：US15224474

申请日：2016-07-29

Applicant: Arcturus Therapeutics, Inc.

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

IPC: A61K31/713 ,  C12N15/113 ,  A61K47/48 ,  A61K9/00

CPC classification number: A61K47/554 ,  A61K9/0019 ,  A61K31/713 ,  A61K47/549 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2320/32

Abstract: Described herein is a compound having the structure of formula I, II, or III, wherein R comprises a double stranded RNA molecule, and L1, L2, and L3 independently for each occurrence comprise a ligand selected from the group consisting of a carbohydrate, a cholesteryl, or a peptide; a pharmaceutically accepted salt or pharmaceutical composition thereof; and a method of making the compound.

公开(公告)号：US09951002B2

公开(公告)日：2018-04-24

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 -L2-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.

公开(公告)号：US09834510B2

公开(公告)日：2017-12-05

申请号：US15393840

申请日：2016-12-29

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 ethene, or an unsubstituted or substituted arene or heteroarene; Y is a bond, an ethene, or an unsubstituted or substituted arene or heteroarene; Z is S or O; L is a linear or branched alkylene of 1 to 6 carbons; R1 and R2 are independently a linear or branched alkyl or alkenyl of 1 to 18 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：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：US20150232851A1

公开(公告)日：2015-08-20

申请号：US14703016

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 target micro-RNAs. 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 single stranded oligomer. Embodiments include UNA oligomers with phosphorothioate or boranophosphate intermonomer linkages. The UNA oligomers can be used to target micro-RNAs to reduce micro-RNA activity.

Abstract translation: 本发明提供可用于靶向微RNA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，并且可以是单链寡聚物。 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寡聚体可用于靶向微RNA以降低微RNA活性。

公开(公告)号：US09051570B2

公开(公告)日：2015-06-09

申请号：US13652965

申请日：2012-10-16

Applicant: Arcturus Therapeutics, Inc.

Inventor： Jesper Wengel

IPC: C07H21/04 ,  C12N15/113 ,  C07H21/00 ,  C12N15/11

CPC classification number: C12N15/113 ,  C07H21/00 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2320/51

Abstract: This invention is directed to oligomers composed of 2′-3′-seco-nucleomonomers and nucleotides or modified nucleotides, wherein the oligomer can be a microRNA and include from one to five 2′-3′-seco-nucleomonomers. The oligomers can have a sequence that is complementary to a target nucleotide sequence and be used for affecting the regulation of gene expression. Among other things, the oligomers can provide reduced off target effects.

Abstract translation: 本发明涉及由2'-3'-中间核单体和核苷酸或修饰的核苷酸组成的寡聚体，其中所述寡聚物可以是微小RNA并且包括一个至五个2'-3'-中间核单体。 寡聚物可以具有与靶核苷酸序列互补的序列，并用于影响基因表达的调节。 除此之外，低聚物可以提供减少的目标效应。

公开(公告)号：US20250161434A1

公开(公告)日：2025-05-22

申请号：US18969110

申请日：2024-12-04

Applicant: Arcturus Therapeutics, Inc.

Inventor： Sean Michael SULLIVAN ,  Daiki MATSUDA ,  Kiyoshi TACHIKAWA ,  Padmanabh CHIVUKULA ,  Priya Prakash KARMALI ,  Jared Henry DAVIS ,  Yanjie BAO ,  Amit SAGI

IPC: A61K39/215 ,  A61K9/51 ,  A61K38/00 ,  A61K39/00 ,  A61K39/12 ,  A61K47/10 ,  A61K47/20 ,  A61K47/26 ,  C07K14/005 ,  C07K14/18 ,  C12N7/00 ,  C12N15/86

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

公开(公告)号：US20250000957A1

公开(公告)日：2025-01-02

申请号：US18690408

申请日：2022-09-15

Applicant: Arcturus Therapeutics, Inc.

Inventor： Ramon DIAZ TRELLES ,  Kiyoshi TACHIKAWA ,  Padmanabh CHIVUKULA

IPC: A61K38/51 ,  A61K48/00 ,  A61P3/00 ,  C12N9/88 ,  C12N15/88

Abstract: Phenylketonuria (PKU) is caused by a mutation in the phenylalanine hydroxylase (P AH) gene in the liver, Provided herein are compositions and methods for treating phenylketonuria (PKU) and related disorders. Polynucleotides for expressing bacterial and plant-derived phenylalanine ammonia lyase (PAL) are provided herein. Also provided herein are methods of treating PKU and related disorders that include administration of polynucleotides encoding PAL proteins.