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公开(公告)号：US11434485B2
公开(公告)日：2022-09-06
申请号：US16980927
申请日：2019-03-14
Applicant: Arcturus Therapeutics, Inc.
Inventor： Zoya Ignatova , Andrew Torda , Marco Matthies
Abstract: A synthetic transfer RNA with an extended anticodon loop. 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：US20220096520A1
公开(公告)日：2022-03-31
申请号：US17311271
申请日：2019-12-06
Applicant: Arcturus Therapeutics, Inc.
Inventor： Carlos G. Perez-Garcia , Padmanabh Chivukula , Kiyoshi Tachikawa
IPC: A61K31/7105 , C12Q1/37 , C07K14/435
Abstract: The present disclosure provides a modified human protein having improved in vivo stability. The modified human protein has been altered from a wild-type human protein at either at least one ubiquitination site, at the signal peptide portion, or both. The protein of the disclosure can be produced from a codon optimized mRNA suitable for administration to a patient suffering from deficiency of the wild-type protein, wherein upon administration of the mRNA to the patient, the modified protein of the disclosure is expressed in the patient in therapeutically effective amounts.
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公开(公告)号：US20210317468A1
公开(公告)日：2021-10-14
申请号：US17239369
申请日：2021-04-23
Applicant: Arcturus Therapeutics, Inc.
Inventor： Pattraranee Limphong , Carlos G. Perez-Garcia , Kiyoshi Tachikawa , Padmanabh Chivukula , Arisa Cale , Angel I-Jou Leu , Jared Davis
IPC: C12N15/82 , C12N15/52 , C07K14/805 , C07K14/775 , C07K14/75 , C07K14/47 , C07K14/415 , C07K14/505
Abstract: This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5′ UTRs, where a 5′ UTR may be expressed by a gene of a plant. In some embodiments, a 5′ UTR may be expressed by a gene of a member of Arabidopsis genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.
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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：US10980895B2
公开(公告)日：2021-04-20
申请号：US16527955
申请日：2019-07-31
Applicant: Arcturus Therapeutics, Inc.
Inventor： Joseph E. Payne , Padmanabh Chivukula , Priya Karmali , Steven P. Tanis
IPC: C07C333/04 , A61K48/00 , A61K9/51 , A61K47/20 , A61P31/00
Abstract: What is described is a compound of formula I consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons; L1 and L2 are the same or different, each a linear alkylene or alkenylene consisting of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.
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Patent Agency Ranking
公开(公告)号：US10815463B2
公开(公告)日：2020-10-27
申请号：US15806096
申请日：2017-11-07
Applicant: Arcturus Therapeutics, Inc.
Inventor： Padmanabh Chivukula , Luigi Warren , Kiyoshi Tachikawa , Joseph E. Payne
IPC: A61K48/00 , C07H21/02 , C07H21/04 , C12N9/10 , C07K14/46 , C07K14/47 , C07K14/505 , C07K14/52 , C07K14/575 , C07K14/705 , C07K14/745 , C07K14/805 , C07K14/81 , C12N9/02 , C12N9/12 , C12N9/44 , C12N9/64 , C12N9/88 , C07K14/445 , A61K39/00
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.
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公开(公告)号：US20200297634A1
公开(公告)日：2020-09-24
申请号：US16823212
申请日：2020-03-18
Applicant: Arcturus Therapeutics, Inc.
Inventor： Priya KARMALI , Padmanabh Chivukula , Joseph E. Payne , Yanjie Bao
Abstract: A method of producing a lipid-encapsulated RNA nanoparticle, comprising the steps a) flowing an aqueous solution comprising an RNA through a 1st tube having an inner diameter (ID) of between about 0.1″ and 0.132″; b) flowing an ethanol solution comprising lipids through a 2nd tube having an ID of between about 0.005″ and 0.02″ at one third the flow rate of the aqueous solution through the 1st tube, wherein the lipids comprise a cationic lipid; and c) mixing the ethanol solution with the aqueous solution by flowing the ethanol solution and the aqueous solution into a mixing module consisting of the 2nd tube perpendicularly joined to the 1st tube; wherein the mixing produces an output solution flowing in the 1st tube comprising a turbulent flow of the RNA and the lipids in between about 10% to 75% ethanol v/v, and wherein the lipid-encapsulated RNA nanoparticles have a bilayer structure.
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公开(公告)号：US20200040026A1
公开(公告)日：2020-02-06
申请号：US16600336
申请日：2019-10-11
Applicant: c/o Arcturus Therapeutics, Inc.
Inventor： Padmanabh CHIVUKULA , Steven P. Tanis , Joseph E. Payne
IPC: C07H19/16 , C07H19/20 , C07H19/207 , C07H21/02 , A61K31/7125
Abstract: What is described is a trinucleotide cap analog comprising m7G(5′)p3-N1pN2 for increased efficiency of in vitro transcription of m7G(5′)p3-RNA, wherein m7G is N7-methylguanosine or analog, (5′)p3 is a 5′,5′-triphosphate bridge, and N1 or N2 or both ribonucleotide analogs linked to each other by a phosphate, p, and wherein the trinucleotide cap analog increases the efficiency of in vitro transcription.
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公开(公告)号：US10519447B2
公开(公告)日：2019-12-31
申请号：US15563579
申请日：2016-04-01
Applicant: Arcturus Therapeutics, Inc.
Inventor： Padmanabh Chivukula , Kiyoshi Tachikawa , Joseph E. Payne
IPC: C12N15/113 , A61P9/00 , A61P31/14 , A61P25/00
Abstract: This invention provides UNA oligomers for regulating the expression of a target gene. The UNA oligomers contain UNA monomer linkers, and may contain one or more nucleotides modified with a 2′-O-methyl group, one or more nucleotides modified with a 2′-deoxy-2′-fluoro group, and one or more phosphorothioate or chiral phosphorothioate intermonomer linkages. UNA oligomers can be used as active agents for preventing or treating disease.
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公开(公告)号：US20190388562A1
公开(公告)日：2019-12-26
申请号：US16527955
申请日：2019-07-31
Applicant: Arcturus Therapeutics, Inc.
Inventor： Joseph E. PAYNE , Padmanabh CHIVUKULA , Priya KARMALI , Steven P. TANIS
IPC: A61K48/00 , A61K47/20 , C07C333/04 , A61K9/51
Abstract: What is described is a compound of formula I consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons; L1 and L2 are the same or different, each a linear alkylene or alkenylene consisting of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.
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