JP6029826B2 - 補因子の非存在下において凝固活性を有する第ix因子変異体および出血性疾患の処置のためのその使用 - Google Patents

補因子の非存在下において凝固活性を有する第ix因子変異体および出血性疾患の処置のためのその使用 Download PDF

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JP6029826B2
JP6029826B2 JP2011520375A JP2011520375A JP6029826B2 JP 6029826 B2 JP6029826 B2 JP 6029826B2 JP 2011520375 A JP2011520375 A JP 2011520375A JP 2011520375 A JP2011520375 A JP 2011520375A JP 6029826 B2 JP6029826 B2 JP 6029826B2
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factor
amino acid
viii
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activity
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JP2011528913A5 (enExample
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ザイフリート,エアハルト
シュットルンプ,ヨルク
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DRK Blutspendedienst Baden Wuerttermberg Hessen gGmbH
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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
    • 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)
    • 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
    • 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)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/948Hydrolases (3) acting on peptide bonds (3.4)
    • G01N2333/95Proteinases, i.e. endopeptidases (3.4.21-3.4.99)
    • G01N2333/964Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue
    • G01N2333/96425Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals
    • G01N2333/96427Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general
    • G01N2333/9643Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general with EC number
    • G01N2333/96433Serine endopeptidases (3.4.21)
    • G01N2333/96441Serine endopeptidases (3.4.21) with definite EC number
    • G01N2333/9645Factor IX (3.4.21.22)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/22Haematology
    • G01N2800/224Haemostasis or coagulation

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Food Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2011520375A 2008-07-28 2009-07-28 補因子の非存在下において凝固活性を有する第ix因子変異体および出血性疾患の処置のためのその使用 Active JP6029826B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP08013561.9 2008-07-28
EP08013561A EP2149603A1 (en) 2008-07-28 2008-07-28 Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders
PCT/EP2009/005465 WO2010012451A1 (en) 2008-07-28 2009-07-28 Factor ix variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders

Publications (3)

Publication Number Publication Date
JP2011528913A JP2011528913A (ja) 2011-12-01
JP2011528913A5 JP2011528913A5 (enExample) 2012-09-20
JP6029826B2 true JP6029826B2 (ja) 2016-11-24

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JP2011520375A Active JP6029826B2 (ja) 2008-07-28 2009-07-28 補因子の非存在下において凝固活性を有する第ix因子変異体および出血性疾患の処置のためのその使用

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US (2) US10125357B2 (enExample)
EP (6) EP2149603A1 (enExample)
JP (1) JP6029826B2 (enExample)
KR (2) KR101947424B1 (enExample)
CN (1) CN102112608B (enExample)
AU (1) AU2009275391B2 (enExample)
CA (2) CA2729501C (enExample)
ES (3) ES2441167T3 (enExample)
WO (1) WO2010012451A1 (enExample)

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HUE067487T2 (hu) 2008-09-15 2024-10-28 Uniqure Biopharma B V IX-es faktor polipeptid mutánsa, alkalmazásai és eljárás az elõállítására
TWI595004B (zh) 2010-11-03 2017-08-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
AU2012222366A1 (en) 2011-03-02 2013-08-01 Novo Nordisk A/S Coagulation factor-targeting to TLT-1 on activated platelets
US10656167B2 (en) 2011-07-25 2020-05-19 Bioverativ Therapeutics Inc. Assays to monitor bleeding disorders
CN103173482A (zh) * 2011-12-22 2013-06-26 武汉科技大学 野生型和突变体hFⅨ毕赤酵母表达载体及构建方法和应用
EP2666782A1 (en) * 2012-05-22 2013-11-27 Imnate Sarl Coagulation factor VIII with reduced immunogenicity.
US20150337284A1 (en) * 2012-10-29 2015-11-26 Regents Of The University Of Minnesota Factor ix variants
CN103060366A (zh) * 2012-12-25 2013-04-24 武汉科技大学 人凝血因子ⅸ突变体毕赤酵母表达载体及构建方法和应用
LT2956477T (lt) 2013-02-15 2021-02-10 Bioverativ Therapeutics Inc. Optimizuotas faktoriaus viii genas
US9651567B2 (en) * 2013-05-14 2017-05-16 Cgt Enterprises, Llc Test strips for determining coagulation factor activities
EP2881463A1 (en) 2013-12-09 2015-06-10 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders
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
EP3325010B1 (en) 2015-07-23 2023-06-21 The Regents of The University of California Antibodies to coagulation factor xia and uses thereof
PL3411478T3 (pl) 2016-02-01 2022-10-03 Bioverativ Therapeutics Inc. Geny zoptymalizowanego czynnika VIII
JP7764129B2 (ja) 2017-07-10 2025-11-05 ユニキュアー アイピー ビー.ブイ. ヒトにおけるaav遺伝子療法のための手段及び方法
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
GB201817810D0 (en) * 2018-10-31 2018-12-19 Ucl Business Plc Modified factor IX polypeptides
WO2020104480A1 (en) * 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
WO2020187969A1 (en) * 2019-03-19 2020-09-24 CSL Behring Lengnau AG Factor ix variants and uses thereof in therapy
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
EP4423119A1 (en) 2021-10-27 2024-09-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
US20250242056A1 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response

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US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
US6531298B2 (en) * 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
WO2001085198A1 (en) 2000-05-10 2001-11-15 Novo Nordisk A/S tHARMACEUTICAL COMPOSITION COMPRISING A FACTOR VIIA AND A FACTOR XIII
US7176288B2 (en) * 2000-09-13 2007-02-13 Novo Nordisk Healthcare A/G Human coagulation factor VII variants
AU2002226028A1 (en) 2000-11-14 2002-05-27 Board Of Regents, Unversity Of Texas Systems Mutant human factor ix with an increased resistance to inhibition by heparin
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CA2729501C (en) 2019-04-02
EP2390323B1 (en) 2013-11-27
CN102112608B (zh) 2013-11-20
ES2753638T3 (es) 2020-04-13
KR101768896B1 (ko) 2017-08-17
KR20110036071A (ko) 2011-04-06
US10125357B2 (en) 2018-11-13
EP2318526A1 (en) 2011-05-11
CA2729501A1 (en) 2010-02-04
US10883097B2 (en) 2021-01-05
CA3033578C (en) 2022-07-26
KR101947424B1 (ko) 2019-02-13
ES2628688T3 (es) 2017-08-03
US20190017039A1 (en) 2019-01-17
ES2441167T3 (es) 2014-02-03
EP2767587A3 (en) 2014-11-26
EP2767587A2 (en) 2014-08-20
CN102112608A (zh) 2011-06-29
AU2009275391B2 (en) 2015-01-22
EP3623470A3 (en) 2020-07-22
EP2767587B1 (en) 2017-04-05
EP3623470A2 (en) 2020-03-18
EP2149603A1 (en) 2010-02-03
EP3241899A1 (en) 2017-11-08
JP2011528913A (ja) 2011-12-01
US20110217284A1 (en) 2011-09-08
KR20170096210A (ko) 2017-08-23
AU2009275391A1 (en) 2010-02-04
WO2010012451A1 (en) 2010-02-04
EP3241899B1 (en) 2019-08-21
EP2390323A2 (en) 2011-11-30
CA3033578A1 (en) 2010-02-04
EP2390323A3 (en) 2012-01-11

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