JP2023540562A - Ｒｎａをキャッピングするための組成物および方法 - Google Patents

Ｒｎａをキャッピングするための組成物および方法 Download PDF

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JP2023540562A
JP2023540562A JP2023515146A JP2023515146A JP2023540562A JP 2023540562 A JP2023540562 A JP 2023540562A JP 2023515146 A JP2023515146 A JP 2023515146A JP 2023515146 A JP2023515146 A JP 2023515146A JP 2023540562 A JP2023540562 A JP 2023540562A
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motif
mrna sequence
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end region
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JP2023540562A5 (enExample
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チェン，クリストファー
ラジーブ，カラントッタチル
レイス，キャロライン
ワーン，クイ
サンジーブ，アトゥール
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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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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1247—DNA-directed RNA polymerase (2.7.7.6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07006—DNA-directed RNA polymerase (2.7.7.6)
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2023515146A 2020-09-04 2021-09-03 Ｒｎａをキャッピングするための組成物および方法 Pending JP2023540562A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063074993P 2020-09-04 2020-09-04
US63/074,993 2020-09-04
PCT/US2021/049158 WO2022051677A1 (en) 2020-09-04 2021-09-03 Compositions and methods for capping rnas

Publications (2)

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JP2023540562A true JP2023540562A (ja) 2023-09-25
JP2023540562A5 JP2023540562A5 (enExample) 2024-09-11

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JP2023515146A Pending JP2023540562A (ja) 2020-09-04 2021-09-03 Ｒｎａをキャッピングするための組成物および方法

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US (2) US12264175B2 (enExample)
EP (1) EP4208549A4 (enExample)
JP (1) JP2023540562A (enExample)
CN (1) CN116368226A (enExample)
WO (1) WO2022051677A1 (enExample)

Cited By (1)

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Publication number Priority date Publication date Assignee Title
JP2025168238A (ja) * 2024-04-26 2025-11-07 ベイジン ユーケアケチュアン ファーマシューティカル テクノロジー カンパニー リミテッド リボース修飾キャップ付き類似体及びその使用

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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
EP4208549A4 (en) 2020-09-04 2025-04-16 Verve Therapeutics, Inc. Compositions and methods for capping RNAs
JP2025507894A (ja) * 2022-03-01 2025-03-21 ヴァーヴ・セラピューティクス，インコーポレーテッド Ｒｎａをキャッピングするための組成物及び方法
CN117430652A (zh) * 2022-07-22 2024-01-23 广州市恒诺康医药科技有限公司 用于rna加帽的环状取代化合物及其应用
WO2024051696A1 (zh) * 2022-09-05 2024-03-14 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
CN116143855B (zh) * 2022-11-08 2023-11-28 江苏申基生物科技有限公司 一种乙烯基膦酸修饰的mRNA帽类似物及其制备方法和应用
CN116143854A (zh) * 2022-11-08 2023-05-23 江苏申基生物科技有限公司 一种核糖环修饰的mRNA帽类似物及其制备方法和应用
KR20240086779A (ko) * 2022-12-05 2024-06-19 경상국립대학교산학협력단 다중 단백질의 발현을 위한 신규 핵산 구조체
AU2024210372C1 (en) * 2023-01-19 2025-11-13 Guangzhou Henovcom Bioscience Co., Ltd. Compound for rna capping and use thereof
CN116375781B (zh) * 2023-03-24 2023-12-29 江苏申基生物科技有限公司 一种tna修饰的帽类似物及其制备方法和应用
CN116554250A (zh) * 2023-05-04 2023-08-08 江苏申基生物科技有限公司 一种pace结构修饰的帽类似物及其应用
CN121311491A (zh) * 2023-06-26 2026-01-09 埃洛诺瓦株式会社 新型修饰三核苷酸加帽衍生物以及引入加帽衍生物的mRNA
US20250026779A1 (en) 2023-06-29 2025-01-23 Nutcracker Therapeutics, Inc. Ethyl modified rna caps and methods of use
WO2025054401A2 (en) * 2023-09-06 2025-03-13 Trilink Biotechnologies, Llc Cap analogs and methods of use thereof
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
WO2025137486A1 (en) * 2023-12-22 2025-06-26 Helix Nanotechnologies Inc Cap compounds and rnas comprising the same
CN120665128A (zh) * 2023-12-29 2025-09-19 财团法人工业技术研究院 化合物、药物组合物、用于加帽rna转录物的试剂盒、以及体外转录的方法
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
EP4722375A1 (en) * 2024-10-07 2026-04-08 ExploRNA Therapeutics Sp. z o. o. Efficient in vitro transcription of artificial mrna with highly modified 5' ends

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