IL280973A - Factor ix encoding nucleotides - Google Patents

Factor ix encoding nucleotides

Info

Publication number
IL280973A
IL280973A IL280973A IL28097321A IL280973A IL 280973 A IL280973 A IL 280973A IL 280973 A IL280973 A IL 280973A IL 28097321 A IL28097321 A IL 28097321A IL 280973 A IL280973 A IL 280973A
Authority
IL
Israel
Prior art keywords
factor
encoding nucleotides
nucleotides
encoding
Prior art date
Application number
IL280973A
Other languages
Hebrew (he)
Inventor
Nathwani Amit
Mcintosh Jenny
Original Assignee
Ucl Business Ltd
Nathwani Amit
Mcintosh Jenny
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
Priority claimed from US16/105,583 external-priority patent/US10842885B2/en
Priority claimed from GBGB1813528.5A external-priority patent/GB201813528D0/en
Application filed by Ucl Business Ltd, Nathwani Amit, Mcintosh Jenny filed Critical Ucl Business Ltd
Publication of IL280973A publication Critical patent/IL280973A/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N15/52Genes encoding for enzymes or proenzymes
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21022Coagulation factor IXa (3.4.21.22)
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/007Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
IL280973A 2018-08-20 2021-02-18 Factor ix encoding nucleotides IL280973A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US16/105,583 US10842885B2 (en) 2018-08-20 2018-08-20 Factor IX encoding nucleotides
GBGB1813528.5A GB201813528D0 (en) 2018-08-20 2018-08-20 Factor IX encoding nucleotides
PCT/GB2019/052339 WO2020039183A1 (en) 2018-08-20 2019-08-20 Factor ix encoding nucleotides

Publications (1)

Publication Number Publication Date
IL280973A true IL280973A (en) 2021-04-29

Family

ID=67810978

Family Applications (1)

Application Number Title Priority Date Filing Date
IL280973A IL280973A (en) 2018-08-20 2021-02-18 Factor ix encoding nucleotides

Country Status (11)

Country Link
EP (1) EP3810770A1 (en)
JP (1) JP2021533799A (en)
KR (1) KR20210049129A (en)
CN (1) CN112739818A (en)
AU (1) AU2019323747A1 (en)
BR (1) BR112021002998A2 (en)
CA (1) CA3109579A1 (en)
IL (1) IL280973A (en)
MX (1) MX2021001901A (en)
SG (1) SG11202101560QA (en)
WO (1) WO2020039183A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CA3225312A1 (en) * 2021-06-23 2022-12-29 Inspirar Limited Composition and method for treating hemophilia b
CA3232470A1 (en) * 2021-10-27 2023-05-04 Leah SABIN Compositions and methods for expressing factor ix for hemophilia b therapy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1804839T3 (en) * 2004-09-22 2012-04-10 St Jude Childrens Res Hospital IMPROVED EXPRESSION OF FACTOR IX IN GENTHERAPY VECTORS
TR201813067T4 (en) * 2008-09-15 2018-09-21 Uniqure Biopharma B V The factor IX polypeptide mutant, its uses and a method for producing it.
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
RU2014144881A (en) * 2012-04-17 2016-06-10 Ф. Хоффманн-Ля Рош Аг METHOD FOR EXPRESSION OF POLYEPEPTIDES USING MODIFIED NUCLEIC ACIDS
CN103667346B (en) * 2013-12-17 2016-01-20 扬州大学 A kind of pRBE-HCR-hAAT-hFIXml plasmid and structure thereof and application
EP3160478A4 (en) * 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508025D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP6831779B2 (en) * 2015-06-23 2021-02-17 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia Modified Factor IX and compositions, methods and uses for gene transfer into cells, organs and tissues
CN116732066A (en) * 2016-10-14 2023-09-12 四川至善唯新生物科技有限公司 Preparation and application of high-activity blood coagulation factor IX mutant, recombinant protein and fusion protein

Also Published As

Publication number Publication date
JP2021533799A (en) 2021-12-09
BR112021002998A2 (en) 2021-05-11
CN112739818A (en) 2021-04-30
KR20210049129A (en) 2021-05-04
SG11202101560QA (en) 2021-03-30
MX2021001901A (en) 2021-05-27
AU2019323747A1 (en) 2021-03-04
WO2020039183A1 (en) 2020-02-27
CA3109579A1 (en) 2020-02-27
EP3810770A1 (en) 2021-04-28

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