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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。
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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.
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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.
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公开(公告)号：US11744887B2
公开(公告)日：2023-09-05
申请号：US17196889
申请日：2021-03-09
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 , C07K14/005 , C12N15/86 , A61K47/20 , A61K47/26 , A61K47/10 , A61K9/51 , A61K39/12 , C07K14/18 , C12N7/00 , A61K39/00 , A61K38/00
CPC classification number: A61K39/215 , A61K9/5123 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/1808 , C12N7/00 , C12N15/86 , A61K38/00 , A61K2039/53 , C12N2770/20022 , C12N2770/20034 , C12N2770/36122 , C12N2770/36134 , C12N2830/42 , C12N2830/50
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.
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公开(公告)号：US11685906B2
公开(公告)日：2023-06-27
申请号：US16705102
申请日：2019-12-05
Applicant: Arcturus Therapeutics, Inc.
Inventor： Carlos G. Perez-Garcia , Kiyoshi Tachikawa , Daiki Matsuda , Padmanabh Chivukula
CPC classification number: C12N9/1018 , A61P9/00 , C12Y201/03003 , A61K48/00
Abstract: The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC deficiency wherein upon administration of the mRNA to the patient, the protein of the disclosure is expressed in the patient in therapeutically effective amounts to treat OTC deficiency. The present disclosure also provides codon optimized mRNA sequences encoding wild type human OTC comprising a 5′ UTR derived from a gene expressed by Arabidopsis thaliana for use in treating OTC deficiency in a patient.
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Patent Agency Ranking
公开(公告)号：USRE49233E1
公开(公告)日：2022-10-04
申请号：US16351301
申请日：2019-03-12
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 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.
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公开(公告)号：US20210292768A1
公开(公告)日：2021-09-23
申请号：US17266556
申请日：2019-08-08
Applicant: ARCTURUS THERAPEUTICS, INC.
Inventor： Kiyoshi Tachikawa , Padmanabh Chivukula , Lily Xu , Angel Leu , Marciano Sablad , Rajesh Mukthavaram , Priya Karmali
IPC: C12N15/113 , A61P1/16
Abstract: This disclosure encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of PDGFRB 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.
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公开(公告)号：US20210284974A1
公开(公告)日：2021-09-16
申请号：US17191247
申请日：2021-03-03
Applicant: Arcturus Therapeutics, Inc.
Inventor： Padmanabh Chivukula , Priya Prakash Karmali , Kiyoshi Tachikawa , Suezanne E. Parker , Marciano Rodriguez Sablad , Pattraranee Limphong , Yanjie Bao , Jerel Boyd Lee Vega
IPC: C12N9/10 , C12N15/113 , A61K31/221
Abstract: The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.
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公开(公告)号：US10961535B2
公开(公告)日：2021-03-30
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.
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公开(公告)号：US10604758B2
公开(公告)日：2020-03-31
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
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.
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