AU2023209862A1 - Poly-tailed and poly-capped mrna and uses thereof - Google Patents

Poly-tailed and poly-capped mrna and uses thereof Download PDF

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Publication number
AU2023209862A1
AU2023209862A1 AU2023209862A AU2023209862A AU2023209862A1 AU 2023209862 A1 AU2023209862 A1 AU 2023209862A1 AU 2023209862 A AU2023209862 A AU 2023209862A AU 2023209862 A AU2023209862 A AU 2023209862A AU 2023209862 A1 AU2023209862 A1 AU 2023209862A1
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Australia
Prior art keywords
unsubstituted
substituted
modified
poly
modified mrna
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Pending
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AU2023209862A
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English (en)
Inventor
Abhishek ADITHAM
Hongyu Chen
Jianting GUO
Xiao Wang
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Massachusetts Institute of Technology
Broad Institute Inc
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Massachusetts Institute of Technology
Broad Institute Inc
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Application filed by Massachusetts Institute of Technology, Broad Institute Inc filed Critical Massachusetts Institute of Technology
Publication of AU2023209862A1 publication Critical patent/AU2023209862A1/en
Priority to AU2025252534A priority Critical patent/AU2025252534A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/595—Polyamides, e.g. nylon
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
AU2023209862A 2022-01-18 2023-01-18 Poly-tailed and poly-capped mrna and uses thereof Pending AU2023209862A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025252534A AU2025252534A1 (en) 2022-01-18 2025-10-15 Poly-tailed and poly-capped mRNA and uses thereof

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263300602P 2022-01-18 2022-01-18
US63/300,602 2022-01-18
PCT/US2023/060852 WO2023141474A1 (en) 2022-01-18 2023-01-18 Poly-tailed and poly-capped mrna and uses thereof

Related Child Applications (1)

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AU2025252534A Division AU2025252534A1 (en) 2022-01-18 2025-10-15 Poly-tailed and poly-capped mRNA and uses thereof

Publications (1)

Publication Number Publication Date
AU2023209862A1 true AU2023209862A1 (en) 2024-08-15

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
AU2023209862A Pending AU2023209862A1 (en) 2022-01-18 2023-01-18 Poly-tailed and poly-capped mrna and uses thereof
AU2025252534A Pending AU2025252534A1 (en) 2022-01-18 2025-10-15 Poly-tailed and poly-capped mRNA and uses thereof

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Application Number Title Priority Date Filing Date
AU2025252534A Pending AU2025252534A1 (en) 2022-01-18 2025-10-15 Poly-tailed and poly-capped mRNA and uses thereof

Country Status (8)

Country Link
US (1) US20240392311A1 (https=)
EP (2) EP4466074A1 (https=)
JP (2) JP2025503730A (https=)
KR (2) KR20260006688A (https=)
CN (2) CN121718542A (https=)
AU (2) AU2023209862A1 (https=)
CA (1) CA3248956A1 (https=)
WO (1) WO2023141474A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025519910A (ja) * 2022-06-24 2025-06-26 ザ・ブロード・インスティテュート・インコーポレイテッド キャップされた環状ｒｎａ分子を調製するための組成物及び方法
WO2025076085A1 (en) * 2023-10-02 2025-04-10 Convergence Bio, Inc. Multi-terminus polya polynucleotides
WO2025111430A1 (en) * 2023-11-21 2025-05-30 Prime Medicine, Inc. CHEMICAL MODIFICATIONS IN mRNA POLY(A) TAIL
WO2025147660A2 (en) * 2024-01-04 2025-07-10 Trilink Biotechnologies, Llc Modified rna for increasing protein expression
WO2025175176A1 (en) * 2024-02-14 2025-08-21 Modernatx, Inc. Synthetic nucleic acids and methods of using the same for improved polypeptide production
WO2025180432A1 (zh) * 2024-02-28 2025-09-04 图维生物医药科技(苏州)有限公司 一种用于rna加帽的酶及其反应体系
WO2025217126A1 (en) * 2024-04-09 2025-10-16 Convergence Bio, Inc. Polynucleotides, 3' chain terminating tails, ligation and uses thereof
WO2025217135A1 (en) * 2024-04-09 2025-10-16 Convergence Bio, Inc. Rna ligation methods and products

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA53669A (fr) * 2018-09-20 2021-07-28 Modernatx Inc Compositions et procédés d'administration d'acides nucléiques
BR112021023411A2 (pt) 2019-05-22 2022-02-01 Massachusetts Inst Technology Composições e métodos de rna circular
JP2024518546A (ja) * 2021-05-12 2024-05-01 ザ ブロード インスティテュート，インコーポレーテッド 修飾されたｍＲＮＡ、修飾された非コードＲＮＡ、およびその使用

Also Published As

Publication number Publication date
KR20240144777A (ko) 2024-10-02
EP4737574A2 (en) 2026-05-06
AU2025252534A1 (en) 2025-11-06
EP4466074A1 (en) 2024-11-27
CN119095964A (zh) 2024-12-06
US20240392311A1 (en) 2024-11-28
CA3248956A1 (en) 2023-07-27
CN121718542A (zh) 2026-03-24
JP2025503730A (ja) 2025-02-04
WO2023141474A9 (en) 2023-10-26
JP2026016516A (ja) 2026-02-03
KR20260006688A (ko) 2026-01-13
WO2023141474A1 (en) 2023-07-27

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