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公开(公告)号：US12077754B2
公开(公告)日：2024-09-03
申请号：US17876803
申请日：2022-07-29
Applicant: ARCTURUS THERAPEUTICS, INC.
Inventor： Zoya Ignatova , Andrew Torda , Marco Matthies
CPC classification number: C12N15/11 , A61K48/00 , C12N2310/3513 , C12N2310/531
Abstract: The invention relates to a synthetic transfer RNA with an extended anticodon loop. The invention provides a synthetic suppressor transfer RNA useful for the treatment of a genetic disease like cystic fibrosis associated with a nonsense mutation. The synthetic transfer RNA contains an extended anticodon loop with two consecutive anticodon base triplets configured to base-pair to two consecutive codon base triplets on an mRNA. The first anticodon base triplet or the second anticodon base triplet is configured to base-pair to a stop codon base triplet on the mRNA.
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公开(公告)号：US11938227B2
公开(公告)日：2024-03-26
申请号：US17473063
申请日：2021-09-13
Applicant: Arcturus Therapeutics, Inc.
Inventor： Yanjie Bao , Brenda Clemente , Priya Prakash Karmali
IPC: A61K9/00 , A61K9/51 , A61K31/00 , A61K31/7105
CPC classification number: A61K9/5192 , 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：US20240002815A1
公开(公告)日：2024-01-04
申请号：US18319977
申请日：2023-05-18
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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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：US20220306678A1
公开(公告)日：2022-09-29
申请号：US17619882
申请日：2020-06-17
Applicant: ARCTURUS THERAPEUTICS, INC.
Inventor： Maher ALAYYOUBI , Jared Henry DAVIS
Abstract: Provided herein are methods for purification of RNA from a sample. The methods include obtaining a first sample including double stranded RNA in a loading buffer, loading the sample onto a ceramic hydroxyapatite column, washing the column with wash buffer, and eluting the column with an elution buffer to create an eluate.
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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：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：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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