JP2023523414A - キャッピング化合物、組成物、及びその使用方法 - Google Patents

キャッピング化合物、組成物、及びその使用方法 Download PDF

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JP2023523414A
JP2023523414A JP2022563870A JP2022563870A JP2023523414A JP 2023523414 A JP2023523414 A JP 2023523414A JP 2022563870 A JP2022563870 A JP 2022563870A JP 2022563870 A JP2022563870 A JP 2022563870A JP 2023523414 A JP2023523414 A JP 2023523414A
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nucleic acid
sequence
acid sequence
encoding nucleic
epitope
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JPWO2021216776A5 (https=
JP2023523414A5 (https=
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カリン ヨース
エイミー レイチェル ラパポート
シアラン ダニエル スカラン
レオニド ギトリン
スー－ジーン ホン
アルビン アクピー
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グリットストーン バイオ インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • C07H1/02—Phosphorylation
    • C07H1/04—Introducing polyphosphoric acid radicals
    • 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
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/60—Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6018—Lipids, e.g. in lipopeptides
    • 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
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36134—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
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36141—Use of virus, viral particle or viral elements as a vector
    • C12N2770/36143—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
    • 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
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36151—Methods of production or purification of viral material
    • 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
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36171—Demonstrated in vivo effect

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2022563870A 2020-04-21 2021-04-21 キャッピング化合物、組成物、及びその使用方法 Pending JP2023523414A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063013456P 2020-04-21 2020-04-21
US63/013,456 2020-04-21
US202063020473P 2020-05-05 2020-05-05
US63/020,473 2020-05-05
PCT/US2021/028486 WO2021216776A2 (en) 2020-04-21 2021-04-21 Capping compounds, compositions and methods of use thereof

Publications (3)

Publication Number Publication Date
JP2023523414A true JP2023523414A (ja) 2023-06-05
JPWO2021216776A5 JPWO2021216776A5 (https=) 2024-05-01
JP2023523414A5 JP2023523414A5 (https=) 2024-05-01

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JP2022563870A Pending JP2023523414A (ja) 2020-04-21 2021-04-21 キャッピング化合物、組成物、及びその使用方法

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US (1) US20230303614A1 (https=)
EP (1) EP4138854A4 (https=)
JP (1) JP2023523414A (https=)
KR (1) KR20230015914A (https=)
CN (1) CN115768437A (https=)
AU (1) AU2021260932A1 (https=)
CA (1) CA3173803A1 (https=)
IL (1) IL296855A (https=)
WO (1) WO2021216776A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024534261A (ja) * 2021-08-27 2024-09-18 上海兆維科技発展有限公司 キャップアナログ及びその使用

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102366490B1 (ko) * 2020-10-20 2022-02-23 에스티팜 주식회사 5'-캡핑된 rna 합성용 올리고뉴클레오티드
CA3236959A1 (en) * 2021-11-08 2023-05-11 Sue-Jean HONG Self-amplifying rna compositions and methods of use thereof
US20250108066A1 (en) * 2022-01-27 2025-04-03 Trilink Biotechnologies, Llc Trinucleotide cap analogs and methods of use thereof
CN115260264B (zh) * 2022-02-28 2023-06-09 广州市恒诺康医药科技有限公司 用于rna加帽的化合物及其应用
JP7642260B2 (ja) 2022-02-28 2025-03-10 ▲広▼州市恒▲諾▼康医▲薬▼科技有限公司 Ｒｎａキャッピング用化合物及びその使用
US12084703B2 (en) 2022-05-12 2024-09-10 SunVax mRNA Therapeutics Inc. Synthetic self-amplifying mRNA molecules with secretion antigen and immunomodulator
IL319652A (en) * 2022-10-04 2025-05-01 BioNTech SE RNA structures and their uses
WO2025037891A1 (ko) * 2023-08-14 2025-02-20 에스티팜 주식회사 5'-캡핑된 rna 합성용 올리고뉴클레오티드
CN120082547B (zh) * 2025-05-06 2025-10-03 北京悦康科创医药科技股份有限公司 用于自复制mRNA的加帽类似物及其应用

Citations (5)

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JPH0898700A (ja) * 1990-08-13 1996-04-16 Isis Pharmaceut Inc 遺伝子発現を検出及び変調する糖修飾されたオリゴヌクレオチド
WO2017208191A1 (en) * 2016-06-02 2017-12-07 Glaxosmithkline Biologicals Sa Zika viral antigen constructs
WO2018075827A1 (en) * 2016-10-19 2018-04-26 Arcturus Therapeutics, Inc. Trinucleotide mrna cap analogs
JP2018527015A (ja) * 2015-09-21 2018-09-20 トリリンク バイオテクノロジーズ インコーポレイテッド ５’キャップ付ｒｎａを合成するための組成物および方法
WO2018208856A1 (en) * 2017-05-08 2018-11-15 Gritstone Oncology, Inc. Alphavirus neoantigen vectors

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PE20191345A1 (es) * 2016-11-23 2019-09-30 Gritstone Oncology Inc Administracion viral de neoantigenos
ES2983060T3 (es) * 2017-08-18 2024-10-21 Modernatx Inc Variantes de ARN polimerasa

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JPH0898700A (ja) * 1990-08-13 1996-04-16 Isis Pharmaceut Inc 遺伝子発現を検出及び変調する糖修飾されたオリゴヌクレオチド
JP2018527015A (ja) * 2015-09-21 2018-09-20 トリリンク バイオテクノロジーズ インコーポレイテッド ５’キャップ付ｒｎａを合成するための組成物および方法
WO2017208191A1 (en) * 2016-06-02 2017-12-07 Glaxosmithkline Biologicals Sa Zika viral antigen constructs
WO2018075827A1 (en) * 2016-10-19 2018-04-26 Arcturus Therapeutics, Inc. Trinucleotide mrna cap analogs
WO2018208856A1 (en) * 2017-05-08 2018-11-15 Gritstone Oncology, Inc. Alphavirus neoantigen vectors

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Title
ANASTASIA KHVOROVA ET AL.: "The chemical evolution of oligonucleotide therapies of clinical utility", NATURE BIOTECHNOLOGY, vol. 35, no. 3, JPN6025049552, 27 February 2017 (2017-02-27), pages 238 - 248, ISSN: 0005743257 *
ANDREW J. GEALL ET AL.: "Nonviral delivery of self-amplifying RNA vaccines"", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 109, no. 36, JPN6025003707, 20 August 2012 (2012-08-20), pages 14604 - 14609, ISSN: 0005743256 *
CHANTAL SCHMIT ET AL.: "The effects of 2′- and 3′-alkyl substituents on oligonucleotide hybridization and stability", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, vol. 4, no. 16, JPN6025049551, August 1994 (1994-08-01), pages 1969 - 1974, ISSN: 0005743259 *
N. NISHIZONO ET AL.: "Effects of 2'-O-(trifluoromethyl)adenosine on oligodeoxynucleotide hybridization and nuclease stabil", NUCLEIC ACIDS RESEARCH, vol. 26, no. 22, JPN7025005212, 1 November 1998 (1998-11-01), pages 5067 - 5072, ISSN: 0005743258 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024534261A (ja) * 2021-08-27 2024-09-18 上海兆維科技発展有限公司 キャップアナログ及びその使用
JP7788558B2 (ja) 2021-08-27 2025-12-18 上海兆維科技発展有限公司 キャップアナログ及びその使用

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IL296855A (en) 2022-11-01
WO2021216776A3 (en) 2022-01-06
EP4138854A2 (en) 2023-03-01
AU2021260932A1 (en) 2022-12-01
CA3173803A1 (en) 2021-10-28
KR20230015914A (ko) 2023-01-31
WO2021216776A2 (en) 2021-10-28
CN115768437A (zh) 2023-03-07
US20230303614A1 (en) 2023-09-28
EP4138854A4 (en) 2024-12-25

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