KR20230042005A - 반감기가 연장된 mrna 치료제를 포함하는 lnp 조성물 - Google Patents

반감기가 연장된 mrna 치료제를 포함하는 lnp 조성물 Download PDF

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KR20230042005A
KR20230042005A KR1020237000782A KR20237000782A KR20230042005A KR 20230042005 A KR20230042005 A KR 20230042005A KR 1020237000782 A KR1020237000782 A KR 1020237000782A KR 20237000782 A KR20237000782 A KR 20237000782A KR 20230042005 A KR20230042005 A KR 20230042005A
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seq
polynucleotide
sequence
utr
fragment
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Korean (ko)
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데이비드 리드
루치 자인
알리시아 빅넬
캐롤라인 코러
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모더나티엑스, 인크.
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    • C—CHEMISTRY; METALLURGY
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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
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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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
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    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Microbiology (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)
  • Dispersion Chemistry (AREA)
  • 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)
KR1020237000782A 2020-06-23 2021-06-23 반감기가 연장된 mrna 치료제를 포함하는 lnp 조성물 Withdrawn KR20230042005A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063042822P 2020-06-23 2020-06-23
US63/042,822 2020-06-23
US202163165094P 2021-03-23 2021-03-23
US63/165,094 2021-03-23
US202163165469P 2021-03-24 2021-03-24
US63/165,469 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
KR20230042005A true KR20230042005A (ko) 2023-03-27

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ID=77155849

Family Applications (1)

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KR1020237000782A Withdrawn KR20230042005A (ko) 2020-06-23 2021-06-23 반감기가 연장된 mrna 치료제를 포함하는 lnp 조성물

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=)

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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
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
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

Family Cites Families (22)

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GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
GB2284210B (en) 1993-11-26 1997-11-12 British Tech Group Translational enhancer DNA from a kinesin gene
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
CA2715078C (en) 2007-09-26 2019-07-23 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
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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
CN116194151A (zh) 2023-05-30
IL299196A (en) 2023-02-01
EP4168556A2 (en) 2023-04-26
MX2022016550A (es) 2023-04-10
BR112022025991A2 (pt) 2023-03-14

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