CN116368226A - 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

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Publication number
CN116368226A
CN116368226A CN202180074617.6A CN202180074617A CN116368226A CN 116368226 A CN116368226 A CN 116368226A CN 202180074617 A CN202180074617 A CN 202180074617A CN 116368226 A CN116368226 A CN 116368226A
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motif
mrna sequence
terminal region
independently
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陈建明
卡兰特赫塔西尔·拉杰夫
卡罗琳·瑞斯
王奎
阿萨尔·桑杰夫
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Weihu Medical Co ltd
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Weihu Medical Co ltd
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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)
CN202180074617.6A 2020-09-04 2021-09-03 用于加帽rna的组合物和方法 Pending CN116368226A (zh)

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 (1)

Publication Number Publication Date
CN116368226A true CN116368226A (zh) 2023-06-30

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CN202180074617.6A Pending CN116368226A (zh) 2020-09-04 2021-09-03 用于加帽rna的组合物和方法

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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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP4650358A3 (en) * 2024-04-26 2026-01-14 Beijing Youcare Kechuang Pharmaceutical Technology Co., Ltd. Ribose-modified cap analog and use thereof
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

Family Cites Families (98)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5149782A (en) 1988-08-19 1992-09-22 Tanox Biosystems, Inc. Molecular conjugates containing cell membrane-blending agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
KR930702373A (ko) 1990-11-08 1993-09-08 안토니 제이. 페이네 합성 올리고누클레오티드에 대한 다중 리포터(Reporter)그룹의 첨합
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US6214345B1 (en) 1993-05-14 2001-04-10 Bristol-Myers Squibb Co. Lysosomal enzyme-cleavable antitumor drug conjugates
ATE247128T1 (de) 1993-09-03 2003-08-15 Isis Pharmaceuticals Inc Aminoderivatisierte nukleoside und oligonukleoside
IL112920A (en) 1994-03-07 2003-04-10 Dow Chemical Co Composition comprising a dendritic polymer complexed with at least one unit of biological response modifier and a process for the preparation thereof
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5801155A (en) 1995-04-03 1998-09-01 Epoch Pharmaceuticals, Inc. Covalently linked oligonucleotide minor grove binder conjugates
US5672662A (en) 1995-07-07 1997-09-30 Shearwater Polymers, Inc. Poly(ethylene glycol) and related polymers monosubstituted with propionic or butanoic acids and functional derivatives thereof for biotechnical applications
US6444806B1 (en) 1996-04-30 2002-09-03 Hisamitsu Pharmaceutical Co., Inc. Conjugates and methods of forming conjugates of oligonucleotides and carbohydrates
US5959090A (en) 1996-07-02 1999-09-28 Glen Research Corporation Chemical phosphorylation of oligonucleotides and reactants used therefor
JP2001505194A (ja) 1996-11-05 2001-04-17 ブリストル―マイヤーズ・スクイブ・カンパニー 分枝ペプチド・リンカー
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6335437B1 (en) 1998-09-07 2002-01-01 Isis Pharmaceuticals, Inc. Methods for the preparation of conjugated oligomers
CA2370478A1 (en) 1999-03-24 2000-09-28 Serge L. Beaucage N-acylphosphoramidites and their use in oligonucleotide synthesis
US6395437B1 (en) 1999-10-29 2002-05-28 Advanced Micro Devices, Inc. Junction profiling using a scanning voltage micrograph
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
US8877901B2 (en) 2002-12-13 2014-11-04 Immunomedics, Inc. Camptothecin-binding moiety conjugates
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
HUE029877T2 (en) * 2003-05-30 2017-04-28 Gilead Pharmasset Llc Modified fluorinated nucleoside analogues
ATE502122T1 (de) 2006-06-01 2011-04-15 Trilink Biotechnologies Chemisch modifizierte oligonukleotidprimer zur nukleinsäureamplifikation
CN104072561B (zh) 2007-06-19 2017-12-22 路易斯安那州州立大学及农业机械学院管理委员会 信使rna帽的抗‑反向硫代磷酸类似物的合成和用途
US20110130440A1 (en) 2008-03-26 2011-06-02 Alnylam Pharmaceuticals, Inc. Non-natural ribonucleotides, and methods of use thereof
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
ES3000111T3 (en) 2009-02-13 2025-02-27 Immunomedics Inc Intermediates for preparing conjugates with an intracellularly-cleavable linkage
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
DE19216461T1 (de) * 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
WO2013059475A1 (en) 2011-10-18 2013-04-25 Life Technologies Corporation Alkynyl-derivatized cap analogs, preparation and uses thereof
JP6351577B2 (ja) 2012-05-21 2018-07-04 アジレント・テクノロジーズ・インクＡｇｉｌｅｎｔ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． 組成物及びオリゴヌクレオチドをコンジュゲートする方法
PL2922554T3 (pl) * 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
EP3155129B1 (en) 2014-06-10 2019-01-16 CureVac AG Method for enhancing rna production
EP3274454B1 (en) 2015-03-25 2021-08-25 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
CN120842296A (zh) 2015-09-21 2025-10-28 垂林克生物技术有限公司 用于合成5’-加帽rna的组合物和方法
SI3362461T1 (sl) 2015-10-16 2022-05-31 Modernatx, Inc. Analogi kape MRNA z modificirano fosfatno povezavo
WO2017066793A1 (en) * 2015-10-16 2017-04-20 Modernatx, Inc. Mrna cap analogs and methods of mrna capping
WO2017066797A1 (en) * 2015-10-16 2017-04-20 Modernatx, Inc. Trinucleotide mrna cap analogs
ES2763822T3 (es) 2015-12-09 2020-06-01 Novartis Ag Análisis exento de marcas de la eficacia de la adición de caperuzas al arn utilizando rnasa h, sondas y cromatografía líquida/espectrometría de masas
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
US20220204978A1 (en) 2019-04-16 2022-06-30 The Regents Of The University Of California Protein translational control
GB2597423B (en) 2019-05-10 2024-08-14 New England Biolabs Inc Chemical capping for template switching
WO2021021677A1 (en) 2019-07-26 2021-02-04 The Regents Of The University Of California Control of mammalian gene dosage using crispr
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
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
GB2613225B (en) 2020-03-04 2024-01-03 Verve Therapeutics Inc Compositions and methods for targeted RNA delivery
CA3176844A1 (en) 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
EP4208549A4 (en) 2020-09-04 2025-04-16 Verve Therapeutics, Inc. Compositions and methods for capping RNAs
US20240175005A1 (en) 2021-03-31 2024-05-30 Modernatx, Inc. PURIFICATION AND RECYCLING OF mRNA NUCLEOTIDE CAPS
US20250108066A1 (en) 2022-01-27 2025-04-03 Trilink Biotechnologies, Llc Trinucleotide cap analogs and methods of use thereof

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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