JP2025507894A - Ｒｎａをキャッピングするための組成物及び方法 - Google Patents

Ｒｎａをキャッピングするための組成物及び方法 Download PDF

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JP2025507894A
JP2025507894A JP2024552144A JP2024552144A JP2025507894A JP 2025507894 A JP2025507894 A JP 2025507894A JP 2024552144 A JP2024552144 A JP 2024552144A JP 2024552144 A JP2024552144 A JP 2024552144A JP 2025507894 A JP2025507894 A JP 2025507894A
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mrna sequence
och
motif
independently
end region
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JPWO2023167880A5 (https=
JP2025507894A5 (https=
Inventor
チェン，クリストファー
ラジーブ，カラントッタチル・ジー
レイス，キャロライン
ワーン，クイ
ライプハイマー，ジェイ
Original Assignee
ヴァーヴ・セラピューティクス，インコーポレーテッド
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Publication of JP2025507894A publication Critical patent/JP2025507894A/ja
Publication of JPWO2023167880A5 publication Critical patent/JPWO2023167880A5/ja
Publication of JP2025507894A5 publication Critical patent/JP2025507894A5/ja
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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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04002—Adenine deaminase (3.5.4.2)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2024552144A 2022-03-01 2023-02-28 Ｒｎａをキャッピングするための組成物及び方法 Pending JP2025507894A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263315323P 2022-03-01 2022-03-01
US63/315,323 2022-03-01
US202363485753P 2023-02-17 2023-02-17
US63/485,753 2023-02-17
PCT/US2023/014183 WO2023167880A2 (en) 2022-03-01 2023-02-28 Compositions and methods for capping rnas

Publications (3)

Publication Number Publication Date
JP2025507894A true JP2025507894A (ja) 2025-03-21
JPWO2023167880A5 JPWO2023167880A5 (https=) 2026-03-10
JP2025507894A5 JP2025507894A5 (https=) 2026-03-10

Family

ID=87884204

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024552144A Pending JP2025507894A (ja) 2022-03-01 2023-02-28 Ｒｎａをキャッピングするための組成物及び方法

Country Status (5)

Country Link
US (1) US20250084409A1 (https=)
EP (1) EP4486355A4 (https=)
JP (1) JP2025507894A (https=)
CN (1) CN119137271A (https=)
WO (1) WO2023167880A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4733306A1 (en) * 2023-06-26 2026-04-29 Elonova Co., Ltd. Novel modified trinucleotide capping derivative, and mrna into which capping derivative is introduced
IL326531A (en) * 2023-09-06 2026-04-01 Trilink Biotechnologies Llc Cap analogues and methods of their use
CN116987137B (zh) * 2023-09-26 2024-01-02 江苏申基生物科技有限公司 一种加帽化合物及其在mRNA加帽中的应用
WO2025168058A1 (zh) * 2024-02-08 2025-08-14 江苏申基生物科技有限公司 新的mRNA加帽化合物及其应用
WO2026009929A1 (ja) * 2024-07-03 2026-01-08 富士フイルム株式会社 化合物、ｒｎａ合成開始剤、およびそれらの利用
CN119306779A (zh) * 2024-12-13 2025-01-14 上海科泽永欣生物科技有限公司 一种含r构型的起始加帽寡核苷酸引物及其合成方法

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2964690T3 (es) * 2015-09-21 2024-04-09 Trilink Biotechnologies Llc Método para sintetizar ARN con caperuza 5'
US11866754B2 (en) * 2015-10-16 2024-01-09 Modernatx, Inc. Trinucleotide mRNA cap analogs
WO2017098468A1 (en) * 2015-12-09 2017-06-15 Novartis Ag Label-free analysis of rna capping efficiency using rnase h, probes and liquid chromatography/mass spectrometry
PL415967A1 (pl) * 2016-01-29 2017-07-31 Univ Warszawski 5'-tiofosforanowe analogi końca 5' mRNA (kapu), sposób ich otrzymywania i zastosowanie
US10487105B2 (en) * 2016-10-19 2019-11-26 Arcturus Therapeutics, Inc. Trinucleotide MRNA cap analogs
KR20200143391A (ko) * 2018-03-15 2020-12-23 비온테크 알엔에이 파마슈티컬스 게엠베하 5'-캡-트리뉴클레오티드- 또는 고차의 올리고뉴클레오티드 화합물 및 이들의 rna 안정화, 단백질 발현 및 치료에서의 사용
PL432884A1 (pl) * 2020-02-12 2021-08-16 Uniwersytet Warszawski Nowe analogi kapu końca 5' mRNA, cząsteczka RNA je zawierająca, ich zastosowania i sposób syntezy cząsteczki RNA i peptydu
PL248416B1 (pl) * 2020-02-12 2025-12-08 Explorna Therapeutics Spolka Z Ograniczona Odpowiedzialnoscia Nowe analogi kapu końca 5’ mRNA, cząsteczka RNA je zawierająca, ich zastosowania i sposób syntezy cząsteczki RNA i peptydu
CN116368226A (zh) * 2020-09-04 2023-06-30 维乎医疗有限公司 用于加帽rna的组合物和方法
US20250108066A1 (en) * 2022-01-27 2025-04-03 Trilink Biotechnologies, Llc Trinucleotide cap analogs and methods of use thereof

Also Published As

Publication number Publication date
EP4486355A2 (en) 2025-01-08
US20250084409A1 (en) 2025-03-13
CN119137271A (zh) 2024-12-13
WO2023167880A3 (en) 2023-10-12
WO2023167880A2 (en) 2023-09-07
EP4486355A4 (en) 2026-04-22

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