CN116194151A - 包含具有延长的半衰期的mRNA治疗剂的LNP组合物 - Google Patents

包含具有延长的半衰期的mRNA治疗剂的LNP组合物 Download PDF

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CN116194151A
CN116194151A CN202180051902.6A CN202180051902A CN116194151A CN 116194151 A CN116194151 A CN 116194151A CN 202180051902 A CN202180051902 A CN 202180051902A CN 116194151 A CN116194151 A CN 116194151A
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seq
polynucleotide
sequence
utr
fragment
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Chinese (zh)
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D·里德
R·杰恩
A·比克内尔
C·科勒
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ModernaTx Inc
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ModernaTx Inc
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • 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
    • 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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  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (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)
  • Immunology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202180051902.6A 2020-06-23 2021-06-23 包含具有延长的半衰期的mRNA治疗剂的LNP组合物 Pending CN116194151A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063042822P 2020-06-23 2020-06-23
US63/042822 2020-06-23
US202163165094P 2021-03-23 2021-03-23
US63/165094 2021-03-23
US202163165469P 2021-03-24 2021-03-24
US63/165469 2021-03-24
PCT/US2021/038780 WO2021262909A2 (en) 2020-06-23 2021-06-23 Lnp compositions comprising mrna therapeutics with extended half-life

Publications (1)

Publication Number Publication Date
CN116194151A true CN116194151A (zh) 2023-05-30

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Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180051902.6A Pending CN116194151A (zh) 2020-06-23 2021-06-23 包含具有延长的半衰期的mRNA治疗剂的LNP组合物

Country Status (11)

Country Link
US (1) US20230242908A1 (https=)
EP (1) EP4168556A2 (https=)
JP (1) JP2023531511A (https=)
KR (1) KR20230042005A (https=)
CN (1) CN116194151A (https=)
AU (1) AU2021297248A1 (https=)
BR (1) BR112022025991A2 (https=)
CA (1) CA3187261A1 (https=)
IL (1) IL299196A (https=)
MX (1) MX2022016550A (https=)
WO (1) WO2021262909A2 (https=)

Cited By (1)

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CN117603029A (zh) * 2023-11-21 2024-02-27 江苏禾本生化有限公司 一种戊菌唑关键中间体的合成方法

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US20250313830A1 (en) * 2022-03-25 2025-10-09 Modernatx, Inc. Messenger ribonucleic acids with extended half-life
US20250221931A1 (en) * 2022-03-25 2025-07-10 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2023196399A1 (en) * 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023215498A2 (en) * 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism
KR20250047488A (ko) * 2023-09-27 2025-04-04 한미약품 주식회사 변형된 폴리아데닐 서열을 포함하는 핵산 분자
KR20250047490A (ko) * 2023-09-27 2025-04-04 한미약품 주식회사 변형된 폴리아데닐 서열 및 ox40l 단백질 코딩서열을 포함하는 핵산 분자
KR20250047489A (ko) * 2023-09-27 2025-04-04 한미약품 주식회사 변형된 폴리아데닐 서열 및 p53 단백질 코딩서열을 포함하는 핵산 분자
WO2025255199A1 (en) * 2024-06-05 2025-12-11 Modernatx, Inc. Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof
WO2026085234A1 (en) * 2024-10-18 2026-04-23 Modernatx, Inc. 3' untranslated region sequences for extending messenger ribonucleic acids half-life

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WO2020056147A2 (en) * 2018-09-13 2020-03-19 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease
WO2020056239A1 (en) * 2018-09-14 2020-03-19 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
CN118369111A (zh) * 2021-12-08 2024-07-19 摩登纳特斯有限公司 单纯疱疹病毒mRNA疫苗

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WO2020056239A1 (en) * 2018-09-14 2020-03-19 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
CN118369111A (zh) * 2021-12-08 2024-07-19 摩登纳特斯有限公司 单纯疱疹病毒mRNA疫苗

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Cited By (2)

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Publication number Priority date Publication date Assignee Title
CN117603029A (zh) * 2023-11-21 2024-02-27 江苏禾本生化有限公司 一种戊菌唑关键中间体的合成方法
CN117603029B (zh) * 2023-11-21 2025-12-19 江苏禾本生化有限公司 一种戊菌唑关键中间体的合成方法

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US20230242908A1 (en) 2023-08-03
WO2021262909A2 (en) 2021-12-30
WO2021262909A3 (en) 2022-04-21
JP2023531511A (ja) 2023-07-24
AU2021297248A1 (en) 2023-02-02
CA3187261A1 (en) 2021-12-30
IL299196A (en) 2023-02-01
EP4168556A2 (en) 2023-04-26
MX2022016550A (es) 2023-04-10
BR112022025991A2 (pt) 2023-03-14
KR20230042005A (ko) 2023-03-27

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