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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：US20230295081A1
公开(公告)日：2023-09-21
申请号：US17985045
申请日：2022-11-10
Applicant: Arcturus Therapeutics, Inc.
Inventor： Kumar RAJAPPAN , Steven TANIS , Amit SAGI , Priya Prakash KARMALI , Padmanabh CHIVUKULA
IPC: C07C333/04 , C12N15/113 , C12N15/88 , C07C229/04 , C07C327/06 , C07C271/20 , C07C275/16 , C07C229/16 , A61K47/69 , A61K47/54
CPC classification number: C07C333/04 , A61K47/543 , A61K47/6911 , C07C229/04 , C07C229/16 , C07C271/20 , C07C275/16 , C07C327/06 , C12N15/88 , C12N15/113 , C07C2601/14 , C07C2602/22 , C07C2602/24 , C07C2602/28 , C12N2310/14
Abstract: The present disclosure describes compounds of Formula (I) and pharmaceutically acceptable salts 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：US20250049950A1
公开(公告)日：2025-02-13
申请号：US18732954
申请日：2024-06-04
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
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.
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Patent Agency Ranking
公开(公告)号：US20210290752A1
公开(公告)日：2021-09-23
申请号：US17196890
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
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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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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公开(公告)号：US20230323345A1
公开(公告)日：2023-10-12
申请号：US18001571
申请日：2021-06-17
Applicant: Arcturus Therapeutics, Inc.
Inventor： Kiyoshi TACHIKAWA , Angel I-Jou LEU , Padmanabh CHIVUKULA , Priya Prakash KARMALI
IPC: C12N15/113 , A61P3/00
CPC classification number: C12N15/113 , A61P3/00 , C12N2310/11 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/3125 , C12N2310/3231
Abstract: An oligomer comprising a sense strand and an antisense strand that mediates RNA interference against a target RNA sequence having a trinucleotide repeat expansion is provided, wherein the antisense strand is complementary to the target RNA sequence and comprises a sequence having at least 80% identity to the sequence of Formula (I): rGrCrUrGrCrUrGrCX1X2rCrUrGrCrUrGrCrUrG (I), wherein X1 and X2 are each independently selected from the group consisting of 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; 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 oligomers are useful as therapeutics targeting polyglutamine diseases and other diseases stemming from a trinucleotide repeat expansion.
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公开(公告)号：US20220168234A1
公开(公告)日：2022-06-02
申请号：US17473063
申请日：2021-09-13
Applicant: Arcturus Therapeutics, Inc.
Inventor： Yanjie BAO , Brenda CLEMENTE , Priya Prakash KARMALI
IPC: A61K9/51 , A61K31/7105
Abstract: A method of producing lipid-encapsulated RNA nanoparticles includes flowing an aqueous solution comprising an RNA through a 1st tube having a first inner diameter (ID); the RNA comprises from about 6,000 to about 13,000 nucleotides; flowing an ethanol solution comprising lipids through a 2nd tube having a second inner diameter (ID), at a flow rate of about 0.2 to about 1 times relative to the aqueous solution through the 1st tube, the lipids comprise a cationic lipid; and mixing the ethanol solution with the aqueous solution; the first ID and second ID and flow rates through the 1st tube and 2nd tube are selected to produce a shear force sufficiently low to preserve the integrity of the RNA; the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about ethanol, the lipid-encapsulated RNA nanoparticles having a bilayer structure.
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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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