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公开(公告)号：US20240115691A1
公开(公告)日：2024-04-11
申请号：US18345893
申请日：2023-06-30
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 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/18 , C12N7/00 , C12N15/86
CPC classification number: A61K39/215 , A61K9/5123 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/1808 , C12N7/00 , C12N15/86 , A61K2039/53
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：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.
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公开(公告)号：US20240316090A1
公开(公告)日：2024-09-26
申请号：US18259535
申请日：2021-12-10
Applicant: ARCTURUS THERAPEUTICS, INC. , JANSSEN PHARMACEUTICALS, INC.
Inventor： Ramón Díaz TRELLES , Man Lu LAM , Kiyoshi TACHIKAWA , Priya Prakash KARMALI , Padmanabh CHIVUKULA
IPC: A61K31/7105 , A61K45/06 , A61P31/20 , C12N9/22 , C12N15/90
CPC classification number: A61K31/7105 , A61K45/06 , A61P31/20 , C12N9/22 , C12N15/907
Abstract: Nucleic acid molecules encoding transcription activator like effector nucleases (TALENs) for targeting a hepatitis B virus (HBV) genome are described. Also described are compositions, host cells, lipids, and pharmaceutical compositions containing the TALENs. Methods for treating hepatitis infection, particularly in individuals having chronic HBV infection, using the pharmaceutical compositions of the invention are also described.
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公开(公告)号：US20230227826A1
公开(公告)日：2023-07-20
申请号：US18001574
申请日：2021-06-17
Applicant: Arcturus Therapeutics, Inc. , National University Corporation Tokai National Higher Education and Research System
Inventor： Kiyoshi TACHIKAWA , Angel I-Jou LEU , Padmanabh CHIVUKULA , Priya Prakash KARMALI , Tomoki HIRUNAGI , Kentaro SAHASHI , Masahisa KATSUNO
IPC: C12N15/113 , A61P3/00
CPC classification number: C12N15/113 , A61P3/00 , C12N2310/11 , C12N2320/35 , C12N2310/32 , C12N2310/31
Abstract: A method for inhibiting expression of an mRNA having an expanded trinucleotide repeat region is provided comprising administering an oligomer comprising a sense strand and an antisense strand wherein: a) the antisense strand comprises a sequence of Formula (I): rGrCrUrGrCrUrGrCX1X2rCrUrGrCrUrGrCrUrG (I), wherein X1 and X2 are each independently selected from rA, rU, rG, rC, UNA-A, UNA-U, UNA-G, and UNA-C and wherein at least one of X1 and X2 is a UNA monomer; b) the oligomer comprises a UNA monomer at the first position at the 5′-end of the sense strand; and the sense strand and the antisense strand each independently include 19-29 monomers. The oligomer can be formulated in a lipid delivery vehicle, and can inhibit expression of Atrophin-1, Huntingtin, Ataxin-1, Ataxin-2, Ataxin-3, Ataxin-7, Alpha1A-voltage-dependent calcium channel subunit, TATA-box binding protein (TBP), Androgen Receptor, PP2A-PR55beta, FMR-1 Protein (FMRP), FMR-2 protein, Frataxin, Dystrophy Protein Kinase (DMPK), or Ataxin-8.
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公开(公告)号：US20210060168A1
公开(公告)日：2021-03-04
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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Patent Agency Ranking
公开(公告)号：US20240115692A1
公开(公告)日：2024-04-11
申请号：US18351392
申请日：2023-07-12
Applicant: Arcturus Therapeutics, Inc.
Inventor： Sean Michael SULLIVAN , Daiki MATSUDA , Kiyoshi TACHIKAWA , Padmanabh CHIVUKULA , Priya Prakash KARMALI , Jared Henry DAVIS , Yanjie BAO
IPC: A61K39/215 , A61K9/51 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/18 , C12N7/00 , C12N15/86
CPC classification number: A61K39/215 , A61K9/5123 , A61K39/12 , A61K47/10 , A61K47/20 , A61K47/26 , C07K14/005 , C07K14/1808 , C12N7/00 , C12N15/86 , A61K2039/53
Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic 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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7.发明公开公开(公告)号：US20230159449A1
公开(公告)日：2023-05-25
申请号：US18052505
申请日：2022-11-03
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 , Yihua PEI
IPC: C07C333/04 , A61K9/14 , A61K9/127 , C07D519/00 , A61K47/24
CPC classification number: C07C333/04 , A61K9/14 , A61K9/1272 , C07D519/00 , A61K47/24
Abstract: Lipid formulations that encapsulate messenger RNA (mRNA) are provided herein. The mRNA can be used to express CFTR protein in vitro or in vivo. The lipid formulations can be administered via inhalation to treat cystic fibrosis.
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公开(公告)号：US20210290756A1
公开(公告)日：2021-09-23
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
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：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
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.
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公开(公告)号：US20240309366A1
公开(公告)日：2024-09-19
申请号：US18260096
申请日：2021-12-30
Applicant: Arcturus Therapeutics, Inc. , CONSORCIO CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED, M.P. , INSTITUT D’INVESTIGACIÓ BIOMÉDICA DE GIRONA DR JOSEP TRUETA , UNIVERSITAT DE BARCELONA
Inventor： Ramón Díaz TRELLES , Kiyoshi TACHIKAWA , Priya Prakash KARMALI , Rajesh MUKTHAVARAM , Padmanabh CHIVUKULA , José María MORENO-NAVARRETE , Aleix Gavaldá NAVARRO , José Manuel FERNÁNDEZ-REAL , Marta Giralt OMS , Francesc Villarroya GOMBAU
IPC: C12N15/113 , A61K9/51 , A61K31/713 , A61P3/04
CPC classification number: C12N15/113 , A61K9/5146 , A61K31/713 , A61P3/04
Abstract: Provided herein are compositions and methods for treating metabolic disorders, such as hepatic steatosis, adipose tissue dysfunction, and insulin resistance. Small interfering RNAs (siRNAs) targeting LPS-binding protein (LBP) that include unlocked nucleotides (UNA) and their therapeutic applications for the treatment of metabolic disorders are provided herein.
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