WO2024254546A3 - Particules pseudovirales contenant des systèmes d'expression d'arnm circulaire et leurs méthodes d'utilisation - Google Patents

Particules pseudovirales contenant des systèmes d'expression d'arnm circulaire et leurs méthodes d'utilisation Download PDF

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Publication number
WO2024254546A3
WO2024254546A3 PCT/US2024/033132 US2024033132W WO2024254546A3 WO 2024254546 A3 WO2024254546 A3 WO 2024254546A3 US 2024033132 W US2024033132 W US 2024033132W WO 2024254546 A3 WO2024254546 A3 WO 2024254546A3
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WIPO (PCT)
Prior art keywords
virus
particles
methods
rna molecule
mrna expression
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Ceased
Application number
PCT/US2024/033132
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English (en)
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WO2024254546A2 (fr
Inventor
Samie R. Jaffrey
Mildred UNTI
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Cornell University
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Cornell University
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Publication date
Application filed by Cornell University filed Critical Cornell University
Priority to EP24820191.5A priority Critical patent/EP4724584A2/fr
Publication of WO2024254546A2 publication Critical patent/WO2024254546A2/fr
Publication of WO2024254546A3 publication Critical patent/WO2024254546A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16023—Virus like particles [VLP]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/20011—Coronaviridae
    • C12N2770/20022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention concerne une particule pseudovirale (VLP). La particule pseudovirale contient une molécule d'ARN circulaire comprenant : une première séquence de ligature ; un site d'entrée ribosomique interne (IRES) couplé à une molécule d'ARN codant pour un ou plusieurs peptides, le site d'entrée ribosomique interne couplé à la molécule d'ARN codant pour un ou plusieurs peptides étant positionné en 3' par rapport à la première séquence de ligature ; une seconde séquence de ligature positionnée en 3' du site d'entrée ribosomique interne couplé à la molécule d'ARN codant pour lesdits un ou plusieurs peptides ; et une pluralité d'une ou plusieurs protéines qui peuvent s'auto-assembler en une nanoparticule. Sont également divulguées des compositions et des méthodes de productions et d'utilisation de telles particules pseudovirales.
PCT/US2024/033132 2023-06-08 2024-06-07 Particules pseudovirales contenant des systèmes d'expression d'arnm circulaire et leurs méthodes d'utilisation Ceased WO2024254546A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP24820191.5A EP4724584A2 (fr) 2023-06-08 2024-06-07 Particules pseudovirales contenant des systèmes d'expression d'arnm circulaire et leurs méthodes d'utilisation

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363506997P 2023-06-08 2023-06-08
US63/506,997 2023-06-08

Publications (2)

Publication Number Publication Date
WO2024254546A2 WO2024254546A2 (fr) 2024-12-12
WO2024254546A3 true WO2024254546A3 (fr) 2025-02-13

Family

ID=93796442

Family Applications (1)

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PCT/US2024/033132 Ceased WO2024254546A2 (fr) 2023-06-08 2024-06-07 Particules pseudovirales contenant des systèmes d'expression d'arnm circulaire et leurs méthodes d'utilisation

Country Status (2)

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EP (1) EP4724584A2 (fr)
WO (1) WO2024254546A2 (fr)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236673A1 (fr) * 2018-06-06 2019-12-12 Massachusetts Institute Of Technology Arn circulaire pour la traduction dans des cellules eucaryotes
US20210340542A1 (en) * 2017-06-23 2021-11-04 Cornell University Rna molecules, methods of producing circular rna, and treatment methods
WO2023024500A1 (fr) * 2021-08-27 2023-03-02 Peking University Constructions et procédés de préparation d'arn circulaire
US20230125935A1 (en) * 2020-06-25 2023-04-27 The Board Of Trustees Of The Leland Stanford Junior University Genetic elements driving circular rna translation and methods of use

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210340542A1 (en) * 2017-06-23 2021-11-04 Cornell University Rna molecules, methods of producing circular rna, and treatment methods
WO2019236673A1 (fr) * 2018-06-06 2019-12-12 Massachusetts Institute Of Technology Arn circulaire pour la traduction dans des cellules eucaryotes
US20230125935A1 (en) * 2020-06-25 2023-04-27 The Board Of Trustees Of The Leland Stanford Junior University Genetic elements driving circular rna translation and methods of use
WO2023024500A1 (fr) * 2021-08-27 2023-03-02 Peking University Constructions et procédés de préparation d'arn circulaire

Also Published As

Publication number Publication date
EP4724584A2 (fr) 2026-04-15
WO2024254546A2 (fr) 2024-12-12

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