CN119137271A - 用于加帽rna的组合物和方法 - Google Patents

用于加帽rna的组合物和方法 Download PDF

Info

Publication number
CN119137271A
CN119137271A CN202380034551.7A CN202380034551A CN119137271A CN 119137271 A CN119137271 A CN 119137271A CN 202380034551 A CN202380034551 A CN 202380034551A CN 119137271 A CN119137271 A CN 119137271A
Authority
CN
China
Prior art keywords
och
mrna sequence
motif
independently
terminal region
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
CN202380034551.7A
Other languages
English (en)
Chinese (zh)
Inventor
陈建明
卡兰特赫塔西尔·G·拉杰夫
卡罗琳·瑞斯
王奎
杰伊·利菲摩尔
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.)
Weihu Medical Co ltd
Original Assignee
Weihu Medical Co ltd
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 Weihu Medical Co ltd filed Critical Weihu Medical Co ltd
Publication of CN119137271A publication Critical patent/CN119137271A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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)

Landscapes

  • 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)
CN202380034551.7A 2022-03-01 2023-02-28 用于加帽rna的组合物和方法 Pending CN119137271A (zh)

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 (1)

Publication Number Publication Date
CN119137271A true CN119137271A (zh) 2024-12-13

Family

ID=87884204

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380034551.7A Pending CN119137271A (zh) 2022-03-01 2023-02-28 用于加帽rna的组合物和方法

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
JP2025507894A (ja) 2025-03-21
WO2023167880A3 (en) 2023-10-12
WO2023167880A2 (en) 2023-09-07
EP4486355A4 (en) 2026-04-22

Similar Documents

Publication Publication Date Title
US12264175B2 (en) Compositions and methods for capping RNAS
CN115484932B (zh) 用于靶向rna递送的组合物和方法
CN119137271A (zh) 用于加帽rna的组合物和方法
EP3350333B1 (en) Polynucleotides containing a stabilizing tail region
EP2447274B1 (en) Oligomeric compounds and methods
KR102014061B1 (ko) 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
JP2024530647A (ja) 標的ｒｎａ送達のための組成物および方法
JP2008501693A (ja) 遺伝子調節で使用するための個別に調節された鎖を有する二本鎖組成物
WO2014159990A1 (en) Interferon production using short rna duplexes
EP4623084A1 (en) Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
WO2024121373A1 (en) Antisense oligonucleotides for the treatment of cardiovascular disease
EP4582540A1 (en) Method for producing nucleic acid molecule
CN118076360A (zh) 用于靶向rna递送的组合物和方法
WO2025210011A1 (en) Antisense oligonucleotides for the treatment of liver disease
WO2026010855A1 (en) Modified rna for increasing protein expression
WO2024115635A1 (en) Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
HK1256703B (en) Polynucleotides containing a stabilizing tail region

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination