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WO2026003269A1 - Plate-forme pour arnm - Google Patents

Plate-forme pour arnm

Info

Publication number
WO2026003269A1
WO2026003269A1 PCT/EP2025/068258 EP2025068258W WO2026003269A1 WO 2026003269 A1 WO2026003269 A1 WO 2026003269A1 EP 2025068258 W EP2025068258 W EP 2025068258W WO 2026003269 A1 WO2026003269 A1 WO 2026003269A1
Authority
WO
WIPO (PCT)
Prior art keywords
utr
sequence
rna molecule
seq
variant
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.)
Pending
Application number
PCT/EP2025/068258
Other languages
English (en)
Inventor
Andre Martin FURGER
Alexander John WELCH
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.)
Oxford University Innovation Ltd
Original Assignee
Oxford University Innovation Ltd
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Oxford University Innovation Ltd filed Critical Oxford University Innovation Ltd
Publication of WO2026003269A1 publication Critical patent/WO2026003269A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

L'invention concerne une plateforme pour ARN comprenant une 5'UTR, une 3'UTR et/ou une séquence codant pour un peptide signal présentant chacune des propriétés avantageuses.
PCT/EP2025/068258 2024-06-27 2025-06-27 Plate-forme pour arnm Pending WO2026003269A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB2409284.3 2024-06-27
GBGB2409284.3A GB202409284D0 (en) 2024-06-27 2024-06-27 Mrna platform

Publications (1)

Publication Number Publication Date
WO2026003269A1 true WO2026003269A1 (fr) 2026-01-02

Family

ID=92213332

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2025/068258 Pending WO2026003269A1 (fr) 2024-06-27 2025-06-27 Plate-forme pour arnm

Country Status (2)

Country Link
GB (1) GB202409284D0 (fr)
WO (1) WO2026003269A1 (fr)

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010129310A1 (fr) * 2009-04-27 2010-11-11 Roswell Park Cancer Institute Réactifs et procédés de production de peptides sécrétés bioactifs
WO2016005324A1 (fr) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilisation de séquences d'adn codant pour une séquence poly (a)
WO2016091391A1 (fr) 2014-12-12 2016-06-16 Curevac Ag Molécules d'acides nucléiques artificielles destinées à améliorer l'expression de protéines
WO2016100812A1 (fr) * 2014-12-19 2016-06-23 Moderna Therapeutics, Inc. Modifications terminales de polynucléotides
WO2018213789A1 (fr) * 2017-05-18 2018-11-22 Modernatx, Inc. Arn messager modifié comprenant des éléments d'arn fonctionnels
WO2020074642A1 (fr) 2018-10-11 2020-04-16 Ethris Gmbh Plasmide contenant une séquence codant pour un arnm à queue poly(a) segmentée
WO2022169895A1 (fr) * 2021-02-02 2022-08-11 Geovax, Inc. Constructions virales destinées à être utilisées dans l'amélioration de l'amorçage de lymphocytes t pendant la vaccination

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010129310A1 (fr) * 2009-04-27 2010-11-11 Roswell Park Cancer Institute Réactifs et procédés de production de peptides sécrétés bioactifs
WO2016005324A1 (fr) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilisation de séquences d'adn codant pour une séquence poly (a)
WO2016091391A1 (fr) 2014-12-12 2016-06-16 Curevac Ag Molécules d'acides nucléiques artificielles destinées à améliorer l'expression de protéines
WO2016100812A1 (fr) * 2014-12-19 2016-06-23 Moderna Therapeutics, Inc. Modifications terminales de polynucléotides
WO2018213789A1 (fr) * 2017-05-18 2018-11-22 Modernatx, Inc. Arn messager modifié comprenant des éléments d'arn fonctionnels
WO2020074642A1 (fr) 2018-10-11 2020-04-16 Ethris Gmbh Plasmide contenant une séquence codant pour un arnm à queue poly(a) segmentée
WO2022169895A1 (fr) * 2021-02-02 2022-08-11 Geovax, Inc. Constructions virales destinées à être utilisées dans l'amélioration de l'amorçage de lymphocytes t pendant la vaccination

Non-Patent Citations (19)

* Cited by examiner, † Cited by third party
Title
ALANINE, D. G. W.QUINKERT, D.KUMARASINGHA, R.MEHMOOD, S.DONNELLAN, F. R.MINKAH, N. K.DADONAITE, B.DIOUF, A.GALAWAY, F.SILK, S. E.: "Human Antibodies that Slow Erythrocyte Invasion Potentiate Malaria-Neutralizing Antibodies", CELL, vol. 178, no. 1, 2019, pages 216 - 228
ANDRIES, O., MC CAFFERTY, S., DE SMEDT, S. C., WEISS, R., SANDERS, N. N., & KITADA, T.: "N1-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice", JOURNAL OF CONTROLLED RELEASE, vol. 217, 2015, pages 337 - 344, XP055231071, Retrieved from the Internet <URL:https://doi.org/10.1016/J.JCONREL.2015.08.051> DOI: 10.1016/j.jconrel.2015.08.051
DATABASE GenBank [online] 12 October 2006 (2006-10-12), STRAUSBERG R L ET AL: "Homo sapiens collagen, type VI, alpha 2, mRNA", XP093314191, retrieved from https://www.ncbi.nlm.nih.gov/nuccore/41350922 Database accession no. BC065509.1 *
DATABASE GenBank [online] 15 July 2006 (2006-07-15), STRAUSBERG R L ET AL: "Homo sapiens H2A histone family, member X, mRNA", XP093314197, retrieved from https://www.ncbi.nlm.nih.gov/nuccore/38196985 Database accession no. BC004915.2 *
DATABASE GenBank [online] 25 July 2016 (2016-07-25), KUBO Y: "Homo sapiens mRNA for cystatin C", XP093314202, retrieved from https://www.ncbi.nlm.nih.gov/nuccore/325651799 Database accession no. AB587083.1 *
DENG, Y.-Q.ZHANG, N.-N.ZHANG, Y.-F.ZHONG, X.XU, S.QIU, H.-Y.WANG, T.-C.ZHAO, H.ZHOU, C.ZU, S.-L.: "Lipid nanoparticle-encapsulated mRNA antibody provides long-term protection against SARS-CoV-2 in mice and hamsters", CELL RESEARCH, vol. 32, no. 4, 2022, pages 375 - 382, XP037789509, DOI: 10.1038/s41422-022-00630-0
FANG, E.LIU, X.LI, M.ZHANG, Z.SONG, L.ZHU, B.WU, X.LIU, J.ZHAO, D.LI, Y.: "Advances in COVID-19 mRNA vaccine development", SIGNAL TRANSDUCTION AND TARGETED THERAPY, 2022, Retrieved from the Internet <URL:https://doi.org/10.1038/s41392-022-00950-y>
GLOVER, D. J.LIPPS, H. J.JANS, D. A.: "TOWARDS SAFE, NON-VIRAL THERAPEUTIC GENE EXPRESSION IN HUMANS", NAT REV GENET, vol. 6, no. 4, 2005, pages 299 - 310, XP055821395, DOI: 10.1038/nrg1577
GRIER, A. E., BURLEIGH, S., SAHNI, J., CLOUGH, C. A., CARDOT, V., CHOE, D. C., KRUTEIN, M. C., RAWLINGS, D. J., JENSEN, M. C., SCH: "pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences", MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 5, 2016, pages e306, XP093197520, DOI: 10.1038/mtna.2016.21
GUAN, X.PEI, Y.SONG, J.: "DNA-Based Nonviral Gene Therapy-Challenging but Promising", MOLECULAR PHARMACEUTICS, vol. 21, no. 2, 2024, pages 427 - 453
HOU, X.ZAKS, T.LANGER, R.DONG, Y.: "Lipid nanoparticles for mRNA delivery", NATURE REVIEWS MATERIALS, vol. 6, no. 12, 2021, pages 1078 - 1094, XP037634156, DOI: 10.1038/s41578-021-00358-0
KARIKO, K.BUCKSTEIN, M.NI, H.WEISSMAN, D.: "Suppression of RNA Recognition by Toll-like Receptors: The Impact of Nucleoside Modification and the Evolutionary Origin of RNA", IMMUNITY, vol. 23, no. 2, 2005, pages 165 - 175
KARIKO, K.MURAMATSU, H.WELSH, F. A.LUDWIG, J.KATO, H.AKIRA, S.WEISSMAN, D.: "Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability", MOLECULAR THERAPY, vol. 16, no. 11, 2008, pages 1833 - 1840, XP093147613, Retrieved from the Internet <URL:https://doi.org/10.1038/mt.2008.200> DOI: 10.1038/mt.2008.200
LUNDSTROM, K., VIRAL VECTORS IN GENE THERAPY: WHERE DO WE VIRAL VECTORS IN GENE THERAPY: WHERE DO WE STAND IN 2023?, 2023, Retrieved from the Internet <URL:https://doi.org/10.3390/v15030698>
MUHAMMAD HAMMAD BUTTMUHAMMAD ZAMANABRAR AHMADRAHIMA KHANTAUQEER HUSSAIN MALLHIMOHAMMAD MEHEDI HASANYUSRA HABIB KHANSARA HAFEEZEHAB: "Appraisal for the Potential of Viral and Nonviral Vectors in Gene Therapy: A Review", GENES, vol. 2, no. 1370, 2022, pages 13
MULRONEY, T. E.POYRY, T.YAM-PUC, J. C.RUST, M.HARVEY, R. F.KALMAR, L.HORNER, E.BOOTH, L.FERREIRA, A. P.STONELEY, M.: "N1-methylpseudouridylation of mRNA causes +1 ribosomal frameshifting", NATURE, 2023, pages 1 - 6, Retrieved from the Internet <URL:https://doi.org/10.1038/s41586-023-06800-3>
OWJI, H.NEZAFAT, N.NEGAHDARIPOUR, M.HAJIEBRAHIMI, A.GHASEMI, Y.: "A comprehensive review of signal peptides: Structure, roles, and applications", EUROPEAN JOURNAL OF CELL BIOLOGY, vol. 97, no. 6, 2018, pages 422 - 441, XP093120251, Retrieved from the Internet <URL:https://doi.org/10.1016/J.EJCB.2018.06.003> DOI: 10.1016/j.ejcb.2018.06.003
PORELLO, I.CELLESI, F.: "Frontiers in Bioengineering and Biotechnology", vol. 11, 2023, FRONTIERS MEDIA S.A., article "Intracellular delivery of therapeutic proteins. New advancements and future directions"
TREPOTEC, Z.GEIGER, J.PLANK, C.ANEJA, M. K.RUDOLPH, C.: "Segmented poly(A) tails significantly reduce recombination of plasmid DNA without affecting mRNA translation efficiency or half-life", RNA, vol. 25, no. 4, 2019, pages 507 - 518, XP009517039, DOI: 10.1261/rna.069286.118

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