AU2021297248A1 - LNP compositions comprising mRNA therapeutics with extended half-life - Google Patents

LNP compositions comprising mRNA therapeutics with extended half-life Download PDF

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Publication number
AU2021297248A1
AU2021297248A1 AU2021297248A AU2021297248A AU2021297248A1 AU 2021297248 A1 AU2021297248 A1 AU 2021297248A1 AU 2021297248 A AU2021297248 A AU 2021297248A AU 2021297248 A AU2021297248 A AU 2021297248A AU 2021297248 A1 AU2021297248 A1 AU 2021297248A1
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AU
Australia
Prior art keywords
seq
polynucleotide
sequence
utr
fragment
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.)
Abandoned
Application number
AU2021297248A
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English (en)
Inventor
Alicia BICKNELL
Ruchi Jain
Caroline Kohrer
David Reid
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.)
ModernaTx Inc
Original Assignee
ModernaTx Inc
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Filing date
Publication date
Application filed by ModernaTx Inc filed Critical ModernaTx Inc
Publication of AU2021297248A1 publication Critical patent/AU2021297248A1/en
Abandoned legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1617Organic compounds, e.g. phospholipids, fats
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector 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)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • 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)
AU2021297248A 2020-06-23 2021-06-23 LNP compositions comprising mRNA therapeutics with extended half-life Abandoned AU2021297248A1 (en)

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
AU2021297248A1 true AU2021297248A1 (en) 2023-02-02

Family

ID=77155849

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021297248A Abandoned AU2021297248A1 (en) 2020-06-23 2021-06-23 LNP compositions comprising mRNA therapeutics with extended half-life

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=)

Families Citing this family (10)

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

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP2288336B8 (en) 2008-04-25 2017-03-22 Northwestern University Nanostructures suitable for sequestering cholesterol
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
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
WO2017066797A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Trinucleotide mrna cap analogs
JP7194594B2 (ja) 2016-05-18 2022-12-22 モデルナティエックス インコーポレイテッド 免疫調節ポリペプチドをコードするmRNAの組み合わせ及びその使用
MX2021003015A (es) * 2018-09-13 2021-07-15 Modernatx Inc Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.
JP2022500444A (ja) * 2018-09-14 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. クリグラー−ナジャー症候群の治療のためのウリジン二リン酸グリコシルトランスフェラーゼ1ファミリー、ポリペプチドa1をコードするポリヌクレオチド
AU2021205337A1 (en) * 2020-01-10 2022-07-21 Modernatx, Inc. Methods of making tolerogenic dendritic cells
EP4096720A2 (en) * 2020-01-30 2022-12-07 ModernaTX, Inc. Mrnas encoding metabolic reprogramming polypeptides and uses thereof
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

Also Published As

Publication number Publication date
US20230242908A1 (en) 2023-08-03
WO2021262909A2 (en) 2021-12-30
WO2021262909A3 (en) 2022-04-21
JP2023531511A (ja) 2023-07-24
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
KR20230042005A (ko) 2023-03-27

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MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period