CN102112608B - 在不存在其辅因子的情况下具有凝血活性的因子ix变体及其治疗出血障碍的用途 - Google Patents

在不存在其辅因子的情况下具有凝血活性的因子ix变体及其治疗出血障碍的用途 Download PDF

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CN102112608B
CN102112608B CN2009801296698A CN200980129669A CN102112608B CN 102112608 B CN102112608 B CN 102112608B CN 2009801296698 A CN2009801296698 A CN 2009801296698A CN 200980129669 A CN200980129669 A CN 200980129669A CN 102112608 B CN102112608 B CN 102112608B
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activity
amino acid
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CN102112608A (zh
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E.塞弗里德
J.塞特鲁姆普夫
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DRK Blutspendedienst Baden Wuerttermberg Hessen gGmbH
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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/948—Hydrolases (3) acting on peptide bonds (3.4)
    • G01N2333/95—Proteinases, i.e. endopeptidases (3.4.21-3.4.99)
    • G01N2333/964—Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue
    • G01N2333/96425—Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals
    • G01N2333/96427—Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general
    • G01N2333/9643—Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general with EC number
    • G01N2333/96433—Serine endopeptidases (3.4.21)
    • G01N2333/96441—Serine endopeptidases (3.4.21) with definite EC number
    • G01N2333/9645—Factor IX (3.4.21.22)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/22—Haematology
    • G01N2800/224—Haemostasis or coagulation

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  • Hematology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (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)
CN2009801296698A 2008-07-28 2009-07-28 在不存在其辅因子的情况下具有凝血活性的因子ix变体及其治疗出血障碍的用途 Active CN102112608B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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
EP08013561.9 2008-07-28
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 (2)

Publication Number Publication Date
CN102112608A CN102112608A (zh) 2011-06-29
CN102112608B true CN102112608B (zh) 2013-11-20

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US (2) US10125357B2 (https=)
EP (6) EP2149603A1 (https=)
JP (1) JP6029826B2 (https=)
KR (2) KR101768896B1 (https=)
CN (1) CN102112608B (https=)
AU (1) AU2009275391B2 (https=)
CA (2) CA2729501C (https=)
ES (3) ES2441167T3 (https=)
WO (1) WO2010012451A1 (https=)

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BR112013021661A2 (pt) 2011-03-02 2016-11-22 Novo Nordisk As objetivação de fator de coagulação a tlt-1 sobre plaquetas ativadas
WO2013016454A1 (en) * 2011-07-25 2013-01-31 Biogen Idec Hemophilia 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
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
WO2014183886A1 (en) 2013-05-14 2014-11-20 Struszym, S.L. 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
WO2017015619A1 (en) 2015-07-23 2017-01-26 The Regents Of The University Of California Antibodies to coagulation factor xia and uses thereof
WO2017136358A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
PT3652326T (pt) 2017-07-10 2024-12-26 Uniqure Ip Bv Meios e métodos para terapia génica de aav em humanos
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd 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
CN113573726A (zh) * 2019-03-19 2021-10-29 康诺贝林伦瑙有限公司 因子ix变体及其在治疗中的用途
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
JP7803014B2 (ja) 2019-09-30 2026-01-21 バイオベラティブ セラピューティクス インコーポレイテッド レンチウイルスベクター製剤
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
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
US20230149563A1 (en) 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
WO2025160324A2 (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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WO2007149406A2 (en) * 2006-06-19 2007-12-27 Nautilus Technology Llc Modified coagulation factor ix polypeptides and use thereof for treatment

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ES2753638T3 (es) 2020-04-13
EP2318526A1 (en) 2011-05-11
WO2010012451A1 (en) 2010-02-04
EP2767587A2 (en) 2014-08-20
CN102112608A (zh) 2011-06-29
CA2729501A1 (en) 2010-02-04
CA3033578A1 (en) 2010-02-04
EP3241899B1 (en) 2019-08-21
KR101947424B1 (ko) 2019-02-13
EP2390323A3 (en) 2012-01-11
ES2628688T3 (es) 2017-08-03
KR20170096210A (ko) 2017-08-23
EP2390323B1 (en) 2013-11-27
EP2149603A1 (en) 2010-02-03
US20110217284A1 (en) 2011-09-08
EP2390323A2 (en) 2011-11-30
JP2011528913A (ja) 2011-12-01
CA3033578C (en) 2022-07-26
KR101768896B1 (ko) 2017-08-17
US10883097B2 (en) 2021-01-05
JP6029826B2 (ja) 2016-11-24
CA2729501C (en) 2019-04-02
EP2767587B1 (en) 2017-04-05
EP3623470A2 (en) 2020-03-18
EP3623470A3 (en) 2020-07-22
US20190017039A1 (en) 2019-01-17
EP2767587A3 (en) 2014-11-26
AU2009275391A1 (en) 2010-02-04
US10125357B2 (en) 2018-11-13
ES2441167T3 (es) 2014-02-03
AU2009275391B2 (en) 2015-01-22
KR20110036071A (ko) 2011-04-06
EP3241899A1 (en) 2017-11-08

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