DK2698163T3 - Kodonoptimerede faktor VIII-varianter og syntetisk leverspecifik promotor - Google Patents

Kodonoptimerede faktor VIII-varianter og syntetisk leverspecifik promotor Download PDF

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DK2698163T3
DK2698163T3 DK13192973.9T DK13192973T DK2698163T3 DK 2698163 T3 DK2698163 T3 DK 2698163T3 DK 13192973 T DK13192973 T DK 13192973T DK 2698163 T3 DK2698163 T3 DK 2698163T3
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ser
fviii
thr
glu
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DK13192973.9T
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Amit Nathwani
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Ucl Business Plc
Thrombosis Res Institute
St Jude Children's Res Hospital
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N15/63—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
    • C12N15/86—Viral vectors
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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

Claims (11)

1. Leverspecifik promotor, der omfatter en nukleotidsekvens, der har mindst 90 % identitet med nukleotidsekvensen ifølge SEQ ID NO: 3, når sekvenssammenligningen udføres over den fulde længde af de to sekvenser, hvilken promotor er mindre end 350 basepar lang og er i stand til at styre ekspressionen af en operabelt koblet ekspressionsduelig nukleotidsekvens.
2. Promotor ifølge krav 1, der har mindst 95 % identitet med nukleotidsekvensen ifølge SEQ ID NO: 3.
3. Promotor, der omfatter nukleotidsekvensen ifølge SEQ ID NO: 3.
4. Promotor ifølge et hvilket som helst af kravene 1 til 3, hvor promotoren er mindre end 300 basepar lang.
5. Promotor, der består af nukleotidsekvensen ifølge SEQ ID NO: 3 .
6. Vektor, der omfatter en promotor ifølge et hvilket som helst af kravene 1 til 5.
7. Vektor ifølge krav 6, hvor promotoren er operabelt koblet til en nukleotidsekvens, der koder for et funktionelt faktor VIII-protein.
8. Vektor ifølge krav 7, hvor nukleotidsekvensen, der koder for det funktionelle faktor VIII-protein, omfatter nukleotidsekvensen ifølge SEQ ID NO: 1, SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6 eller SEQ ID NO: 7.
9. Vektor ifølge et hvilket som helst af kravene 6 til 8, hvor vektoren er en virusvektor.
10. Vektor ifølge et hvilket som helst af kravene 6 til 9, hvor vektoren er en rekombinant adenoassocieret virus (rAAV)- vektor .
11. Promotor ifølge et hvilket som helst af kravene 1 til 5 eller vektor ifølge et hvilket som helst af kravene 6 til 10 til anvendelse til behandling af hæmofili, især haemofili A.
DK13192973.9T 2009-07-08 2010-07-08 Kodonoptimerede faktor VIII-varianter og syntetisk leverspecifik promotor DK2698163T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0911870.4A GB0911870D0 (en) 2009-07-08 2009-07-08 Optimised coding sequence and promoter
EP10731905.5A EP2451474B1 (en) 2009-07-08 2010-07-08 Codon-optimized factor viii variants and synthetic liver-specific promoter

Publications (1)

Publication Number Publication Date
DK2698163T3 true DK2698163T3 (da) 2017-12-11

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US (4) US9393323B2 (da)
EP (4) EP3456341A1 (da)
CA (2) CA3003790C (da)
DK (1) DK2698163T3 (da)
ES (2) ES2652241T3 (da)
GB (1) GB0911870D0 (da)
HK (1) HK1248599A1 (da)
LT (1) LT2698163T (da)
NO (1) NO2698163T3 (da)
PL (1) PL2698163T3 (da)
PT (1) PT2698163T (da)
WO (1) WO2011005968A1 (da)

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US20150273082A1 (en) 2015-10-01
CA3003790C (en) 2019-12-31
LT2698163T (lt) 2018-01-10
NO2698163T3 (da) 2018-02-10
EP2698163B1 (en) 2017-09-13
US10709796B2 (en) 2020-07-14
US20130024960A1 (en) 2013-01-24
US9393323B2 (en) 2016-07-19
US20180008726A1 (en) 2018-01-11
PT2698163T (pt) 2017-11-09
EP3456341A1 (en) 2019-03-20
CA3003790A1 (en) 2011-01-13
ES2652241T3 (es) 2018-02-01
WO2011005968A1 (en) 2011-01-13
GB0911870D0 (en) 2009-08-19
US20210000978A1 (en) 2021-01-07
PL2698163T3 (pl) 2018-03-30
EP2451474B1 (en) 2013-12-18
CA2804602C (en) 2022-08-23
CA2804602A1 (en) 2011-01-13
ES2452890T3 (es) 2014-04-03
EP2451474A1 (en) 2012-05-16
HK1248599A1 (zh) 2018-10-19
EP3263126A1 (en) 2018-01-03
EP2698163A1 (en) 2014-02-19
US9764045B2 (en) 2017-09-19

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