JP2025505576A - ｍＲＮＡの発現及び送達システム - Google Patents

ｍＲＮＡの発現及び送達システム Download PDF

Info

Publication number
JP2025505576A
JP2025505576A JP2024545925A JP2024545925A JP2025505576A JP 2025505576 A JP2025505576 A JP 2025505576A JP 2024545925 A JP2024545925 A JP 2024545925A JP 2024545925 A JP2024545925 A JP 2024545925A JP 2025505576 A JP2025505576 A JP 2025505576A
Authority
JP
Japan
Prior art keywords
rna polynucleotide
virus
utr
rna
polynucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024545925A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023154734A5 (https=
JP2025505576A5 (https=
Inventor
デイビッド バーホーベン，
ワイアット アレン ミラー，
バラジ ナラシムハン，
マイケル キンバー，
ダグラス ジョーンズ，
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Iowa State University Research Foundation Inc ISURF
Original Assignee
Iowa State University Research Foundation Inc ISURF
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Iowa State University Research Foundation Inc ISURF filed Critical Iowa State University Research Foundation Inc ISURF
Publication of JP2025505576A publication Critical patent/JP2025505576A/ja
Publication of JPWO2023154734A5 publication Critical patent/JPWO2023154734A5/ja
Publication of JP2025505576A5 publication Critical patent/JP2025505576A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/145—Orthomyxoviridae, e.g. influenza virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005—Wall or coating material
    • A61K9/5021—Organic macromolecular compounds
    • A61K9/5031—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poly(lactide-co-glycolide)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005—Wall or coating material
    • A61K9/5063—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5068—Cell membranes or bacterial membranes enclosing drugs
    • 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
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0693—Tumour cells; Cancer cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • 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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/16011—Orthomyxoviridae
    • C12N2760/16111—Influenzavirus A, i.e. influenza A virus
    • C12N2760/16122—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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/16011—Orthomyxoviridae
    • C12N2760/16111—Influenzavirus A, i.e. influenza A virus
    • C12N2760/16134—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
    • 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
    • C12N2770/38011—Tombusviridae
    • C12N2770/38021—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2770/38011—Tombusviridae
    • C12N2770/38041—Use of virus, viral particle or viral elements as a vector
    • C12N2770/38043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Botany (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024545925A 2022-02-08 2023-02-08 ｍＲＮＡの発現及び送達システム Pending JP2025505576A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263267703P 2022-02-08 2022-02-08
US63/267,703 2022-02-08
PCT/US2023/062194 WO2023154734A2 (en) 2022-02-08 2023-02-08 Mrna expression and delivery systems

Publications (3)

Publication Number Publication Date
JP2025505576A true JP2025505576A (ja) 2025-02-28
JPWO2023154734A5 JPWO2023154734A5 (https=) 2026-02-04
JP2025505576A5 JP2025505576A5 (https=) 2026-02-04

Family

ID=87565058

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024545925A Pending JP2025505576A (ja) 2022-02-08 2023-02-08 ｍＲＮＡの発現及び送達システム

Country Status (7)

Country Link
US (1) US20250236645A1 (https=)
EP (1) EP4476252A4 (https=)
JP (1) JP2025505576A (https=)
CN (1) CN118715326A (https=)
AU (1) AU2023219116A1 (https=)
CA (1) CA3250246A1 (https=)
WO (1) WO2023154734A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119876129A (zh) * 2024-08-16 2025-04-25 中国农业科学院烟草研究所(中国烟草总公司青州烟草研究所) 一种启动子元件及对植物进行基因编辑的方法

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1428886B1 (de) * 2002-12-09 2009-08-05 Celltech GmbH Biotechnologie Verbesserte retrovirale Vektoren für die Gentherapie
US20080171361A1 (en) * 2007-01-11 2008-07-17 The Board Of Regents For Oklahoma State University CAP-Independent Translational Enhancer for Protein Synthesis in Wheat Germ Extract and Transgenic Cereals
US9534234B2 (en) * 2014-04-09 2017-01-03 Dna2.0, Inc. Enhanced nucleic acid constructs for eukaryotic gene expression
EP3551230A1 (en) * 2016-12-08 2019-10-16 CureVac AG Rna for treatment or prophylaxis of a liver disease
CN109971775B (zh) * 2017-12-27 2021-12-28 康码（上海）生物科技有限公司 一种核酸构建物及其调控蛋白合成的方法
FR3081169B1 (fr) * 2018-05-15 2020-06-19 Messenger Biopharma Substitution de la coiffe des arn messagers par deux sequences d'arn introduites a leur extremite 5'
US12264323B2 (en) * 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
IL296617A (en) * 2020-03-20 2022-11-01 Biontech Us Inc Vaccines against the corona virus and methods of use
EP4277641A4 (en) * 2021-02-26 2025-03-19 Duke University Compositions for and methods of improving gene therapy

Also Published As

Publication number Publication date
WO2023154734A2 (en) 2023-08-17
CN118715326A (zh) 2024-09-27
WO2023154734A3 (en) 2023-12-14
EP4476252A2 (en) 2024-12-18
EP4476252A4 (en) 2026-03-18
CA3250246A1 (en) 2023-08-17
US20250236645A1 (en) 2025-07-24
AU2023219116A1 (en) 2024-08-15

Similar Documents

Publication Publication Date Title
JP6745923B2 (ja) 細胞内在化を誘導するためのｃｄ２０結合免疫毒素及びそれを用いる方法
JP2024510421A (ja) Ｖｌｐエンテロウイルスワクチン
CN103319610B (zh) 重组融合蛋白及其制法和用途
AU2016207099A1 (en) Virus-like particle with efficient epitope display
US20110293727A1 (en) Chimeric therapeutics, compositions, and methods for using same
BE1023213B1 (fr) Complexes issus du cytomegalovirus et leurs utilisations
JP2022528298A (ja) リンカー
WO2010042751A2 (en) Chimeric therapeutics, compositions, and methods for using same
CA3136882A1 (fr) Substitution de la coiffe des arn messagers par deux sequences d'arn introduites a leur extremite 5'
EP3302531B1 (en) Immune tolerant and non-immune tolerant elastin-like recombinant peptides and methods of use
JP7069135B2 (ja) アルブミンベースのナノ医薬品を使用するための組成物及び方法
JP2025138811A (ja) 合成ｒｉｇ－ｉ様受容体アゴニスト
US20250236645A1 (en) Mrna expression and delivery systems
US20160000901A1 (en) Compositions and Methods for the Production of Virus-Like Particles
WO2018074558A1 (ja) 複合ポリペプチド単量体、細胞浸透機能を有する当該複合ポリペプチドの単量体の会合体、及び、当該会合体を有効成分とする皮下、皮内、経皮又は筋肉内投与用ノロウイルスコンポーネントワクチン
US20250042962A1 (en) Compositions targeting apoptosis-associated speck-like protein with caspase activation and recruitment domain (asc) and methods of use
CN115960180A (zh) 2019-nCoV S蛋白的突变体及其基因工程化的mRNA和疫苗组合物
WO2025027181A1 (en) Agents and methods for targeted delivery to cells
US20240335555A1 (en) Technologies for preventing or treating infections
CN107513107A (zh) 抗肿瘤融合蛋白及其制法和应用
KR102201154B1 (ko) 폴리글루타메이트-TAT-Cre 융합 단백질의 제조방법
WO2021039873A1 (ja) ウイルスの非構造タンパク質が担持された複合タンパク質単量体、当該単量体の会合体、及び当該会合体を有効成分とするコンポーネントワクチン
CN116444629B (zh) 一种中东呼吸综合征冠状病毒的重组蛋白及疫苗
US20250304623A1 (en) Peptides for intracellular delivery
WO2026027759A1 (en) Agents and methods for targeted delivery to cells

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260127

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20260127