CN115768437A - 加帽化合物、组合物及其使用方法 - Google Patents

加帽化合物、组合物及其使用方法 Download PDF

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Publication number
CN115768437A
CN115768437A CN202180044091.7A CN202180044091A CN115768437A CN 115768437 A CN115768437 A CN 115768437A CN 202180044091 A CN202180044091 A CN 202180044091A CN 115768437 A CN115768437 A CN 115768437A
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sequence
nucleic acid
acid sequence
encoding nucleic
epitope
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CN202180044091.7A
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English (en)
Chinese (zh)
Inventor
K·朱斯
A·R·拉帕波特
C·D·斯卡伦
L·吉特林
S-J·洪
A·阿库派
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Seattle Project Co
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Millstone Biological Co
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    • 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
    • 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
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    • C07H1/02—Phosphorylation
    • C07H1/04—Introducing polyphosphoric acid radicals
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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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  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Biotechnology (AREA)
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  • 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)
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  • 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)
CN202180044091.7A 2020-04-21 2021-04-21 加帽化合物、组合物及其使用方法 Pending CN115768437A (zh)

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 (1)

Publication Number Publication Date
CN115768437A true CN115768437A (zh) 2023-03-07

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CN202180044091.7A Pending CN115768437A (zh) 2020-04-21 2021-04-21 加帽化合物、组合物及其使用方法

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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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116438306A (zh) * 2020-10-20 2023-07-14 St制药株式会社 用于5'-加帽的rna合成的寡核苷酸
CN120082547A (zh) * 2025-05-06 2025-06-03 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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118978560A (zh) * 2021-08-27 2024-11-19 上海兆维科技发展有限公司 一种加帽类似物及其应用
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

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WO2018075827A1 (en) * 2016-10-19 2018-04-26 Arcturus Therapeutics, Inc. Trinucleotide mrna cap analogs
CN108366604A (zh) * 2015-09-21 2018-08-03 垂林克生物技术公司 用于合成5’-加帽rna的组合物和方法
US20190134184A1 (en) * 2016-06-02 2019-05-09 Glaxosmithkline Biologicals, S.A. Zika viral antigen constructs
CN110545810A (zh) * 2016-11-23 2019-12-06 磨石肿瘤生物技术公司 新抗原的病毒递送

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JPH0813274B2 (ja) * 1990-08-13 1996-02-14 アイシス・ファーマシューティカルス・インコーポレーテッド 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
MX2019013259A (es) * 2017-05-08 2020-01-13 Gritstone Oncology Inc Vectores de neoantigeno de alfavirus.
ES2983060T3 (es) * 2017-08-18 2024-10-21 Modernatx Inc Variantes de ARN polimerasa

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CN108366604A (zh) * 2015-09-21 2018-08-03 垂林克生物技术公司 用于合成5’-加帽rna的组合物和方法
US20190134184A1 (en) * 2016-06-02 2019-05-09 Glaxosmithkline Biologicals, S.A. Zika viral antigen constructs
WO2018075827A1 (en) * 2016-10-19 2018-04-26 Arcturus Therapeutics, Inc. Trinucleotide mrna cap analogs
CN110545810A (zh) * 2016-11-23 2019-12-06 磨石肿瘤生物技术公司 新抗原的病毒递送

Non-Patent Citations (2)

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GENLL ANDREW J.，等: "Nonviral delivery of self-amplifying RNA vaccines", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 109, no. 36, 20 August 2012 (2012-08-20), pages 14604 - 14609 *
YAZAN M. ABBAS，等: "Structure of human IFIT1 with capped RNA reveals adaptable mRNA binding and mechanisms for sensing N1 and N2 ribose 2-O methylations", BIOCHEMISTRY, vol. 114, no. 11, 1 March 2017 (2017-03-01), pages 2106, XP055606006, DOI: 10.1073/pnas.1612444114 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116438306A (zh) * 2020-10-20 2023-07-14 St制药株式会社 用于5'-加帽的rna合成的寡核苷酸
CN116438306B (zh) * 2020-10-20 2025-11-11 St制药株式会社 用于5′-加帽的rna合成的寡核苷酸
CN120082547A (zh) * 2025-05-06 2025-06-03 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

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Publication number Publication date
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
US20230303614A1 (en) 2023-09-28
JP2023523414A (ja) 2023-06-05
EP4138854A4 (en) 2024-12-25

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