Patents Assigned to Arcturus Therapeutics, Inc.
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Patent number: 12220455Abstract: 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.Type: GrantFiled: June 30, 2023Date of Patent: February 11, 2025Assignee: Arcturus Therapeutics, Inc.Inventors: Sean Michael Sullivan, Daiki Matsuda, Kiyoshi Tachikawa, Padmanabh Chivukula, Priya Prakash Karmali, Jared Henry Davis, Yanjie Bao, Amit Sagi
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Patent number: 12220485Abstract: 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.Type: GrantFiled: August 28, 2023Date of Patent: February 11, 2025Assignee: Arcturus Therapeutics, Inc.Inventors: Priya Karmali, Padmanabh Chivukula, Joseph E. Payne, Yanjie Bao, Michael Figa, Scott A. Roberts, Andreas Wagner
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Patent number: 12178921Abstract: Methods of preparing lyophilized lipid nanoparticle-nucleic acid compositions are provided. The methods comprise preparing a suspension of lipid nanoparticles with a monosaccharide and one or more excipients selected from thiosulfate, potassium sorbate, sodium benzoate, and iodixanol. Lyophilized lipid nanoparticle-nucleic acid compositions and methods of reconstituting and administering the same are further provided.Type: GrantFiled: August 13, 2021Date of Patent: December 31, 2024Assignee: Arcturus Therapeutics, Inc.Inventors: Amit Sagi, Yanjie Bao, Priya Prakash Karmali
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Patent number: 12083224Abstract: Nucleic acid immunization is achieved by delivering a nucleic acid (NA), e.g., a mRNA or a DNA, encapsulated within a lipid-NA nanoparticle. The NA encodes an immunogenic compound of interest. The lipid-NA nanoparticle is effective for in vivo delivery of NA to a vertebrate cell, including upon administration to a subject. The lipid-NA nanoparticle are incorporated in pharmaceutical compositions for immunizing subjects against various diseases.Type: GrantFiled: April 1, 2019Date of Patent: September 10, 2024Assignee: Arcturus Therapeutics, Inc.Inventors: Padmanabh Chivukula, Priya Karmali, Christine Esau, Jerel Vega, Yanjie Bao, Rajesh Mukthavaram, Samantha Murphy
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Patent number: 12077754Abstract: 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.Type: GrantFiled: July 29, 2022Date of Patent: September 3, 2024Assignee: Arcturus Therapeutics, Inc.Inventors: Zoya Ignatova, Andrew Torda, Marco Matthies
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Patent number: 12070509Abstract: 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.Type: GrantFiled: April 30, 2021Date of Patent: August 27, 2024Assignee: Arcturus Therapeutics, Inc.Inventors: Carlos G. Perez-Garcia, Kiyoshi Tachikawa, Daiki Matsuda, Padmanabh Chivukula, Priya Prakash Karmali, Yanjie Bao, Jerel Boyd Lee Vega, Rajesh Mukthavaram, Amit Sagi
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Patent number: 11939600Abstract: This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human phenylalanine hydroxylase (PAH), or a fragment thereof having PAH activity. The polynucleotide and oligomer molecules are expressible to provide the human PAH or a fragment thereof having PAH activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with phenylketonuria, decreased metabolism of phenylalanine, or increased levels of phenylalanine in a subject.Type: GrantFiled: May 31, 2018Date of Patent: March 26, 2024Assignee: Arcturus Therapeutics, Inc.Inventors: Kiyoshi Tachikawa, Carlos Gustavo Perez-Garcia, Padmanabh Chivukula, Hari Bhaskaran, Sean Christopher Daugherty, Christian W. Cobaugh
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Patent number: 11939363Abstract: 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.Type: GrantFiled: July 14, 2022Date of Patent: March 26, 2024Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Pattraranee Limphong, Kiyoshi Tachikawa, Padmanabh Chivukula, Daiki Matsuda, Arisa Cale
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Patent number: 11938227Abstract: 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.Type: GrantFiled: September 13, 2021Date of Patent: March 26, 2024Assignee: Arcturus Therapeutics, Inc.Inventors: Yanjie Bao, Brenda Clemente, Priya Prakash Karmali
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Publication number: 20230390389Abstract: A nucleic acid vaccine composition comprising one or more of a plasmid-based nucleic acid vaccine and immunotherapy, as well as a lipid formulation, is provided. In addition, the present invention provides a method of enhancing the potency of plasmid-based DNA vaccines and immunotherapies, by formulating a vaccine and/or immunotherapy in a lipid formulation, which is stable when refrigerated or stored frozen, is then delivered to a vaccinee by either needle/syringe, jet injection, or microneedles. The lipid formulation of the present invention comprises one or more lipid excipients selected from 1,2-Distearoyl-sn-glycero-3-phosphocholine, Cholest-5-en-3?-ol, 1,2-Dimyristoyl-rac-glycero-3-methylpolyoxyethlene, and or more symmetric ionizable cationic lipids. The present invention increases vaccine potency dramatically.Type: ApplicationFiled: August 18, 2023Publication date: December 7, 2023Applicants: The Government of the United States, as Represented by the Secretary of the Army, Arcturus Therapeutics, Inc.Inventors: Jay W. HOOPER, Eric M. MUCKER, Padmanabh CHIVUKULA
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Patent number: 11759515Abstract: 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.Type: GrantFiled: March 9, 2021Date of Patent: September 19, 2023Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Sean Michael Sullivan, Daiki Matsuda, Kiyoshi Tachikawa, Padmanabh Chivukula, Priya Prakash Karmali, Jared Henry Davis, Yanjie Bao
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Patent number: 11744887Abstract: 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.Type: GrantFiled: March 9, 2021Date of Patent: September 5, 2023Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Sean Michael Sullivan, Daiki Matsuda, Kiyoshi Tachikawa, Padmanabh Chivukula, Priya Prakash Karmali, Jared Henry Davis, Yanjie Bao, Amit Sagi
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Patent number: 11737979Abstract: 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.Type: GrantFiled: March 18, 2020Date of Patent: August 29, 2023Assignee: Arcturus Therapeutics, Inc.Inventors: Priya Karmali, Padmanabh Chivukula, Joseph E. Payne, Yanjie Bao
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Patent number: 11685921Abstract: 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.Type: GrantFiled: December 20, 2019Date of Patent: June 27, 2023Assignee: Arcturus Therapeutics, Inc.Inventors: Pattraranee Limphong, Kiyoshi Tachikawa, Christine Esau, Padmanabh Chivukula
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Patent number: 11685906Abstract: 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.Type: GrantFiled: December 5, 2019Date of Patent: June 27, 2023Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Carlos G. Perez-Garcia, Kiyoshi Tachikawa, Daiki Matsuda, Padmanabh Chivukula
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Patent number: 11434485Abstract: 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.Type: GrantFiled: March 14, 2019Date of Patent: September 6, 2022Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Zoya Ignatova, Andrew Torda, Marco Matthies
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Patent number: 11407800Abstract: 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.Type: GrantFiled: February 27, 2018Date of Patent: August 9, 2022Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Pattraranee Limphong, Kiyoshi Tachikawa, Padmanabh Chivukula, Daiki Matsuda, Arisa Cale
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Patent number: 11015204Abstract: 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.Type: GrantFiled: May 31, 2018Date of Patent: May 25, 2021Assignee: Arcturus Therapeutics, Inc.Inventors: Pattraranee Limphong, Carlos G. Perez-Garcia, Kiyoshi Tachikawa, Padmanabh Chivukula, Arisa Cale, Angel I-Jou Leu, Jared Davis
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Patent number: 10980895Abstract: 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.Type: GrantFiled: July 31, 2019Date of Patent: April 20, 2021Assignee: Arcturus Therapeutics, Inc.Inventors: Joseph E. Payne, Padmanabh Chivukula, Priya Karmali, Steven P. Tanis
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Patent number: RE49233Abstract: 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.Type: GrantFiled: March 12, 2019Date of Patent: October 4, 2022Assignee: ARCTURUS THERAPEUTICS, INC.Inventors: Joseph E. Payne, Padmanabh Chivukula