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
mutant
activity
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JP2011528913A5 (https=
JP2011528913A (ja
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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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    • 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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  • 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 (https=) 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

Country Status (9)

Country Link
US (2) US10125357B2 (https=)
EP (6) EP2149603A1 (https=)
JP (1) JP6029826B2 (https=)
KR (2) KR101947424B1 (https=)
CN (1) CN102112608B (https=)
AU (1) AU2009275391B2 (https=)
CA (2) CA2729501C (https=)
ES (3) ES2628688T3 (https=)
WO (1) WO2010012451A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TR201813067T4 (tr) 2008-09-15 2018-09-21 Uniqure Biopharma B V Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem.
TWI557135B (zh) 2010-11-03 2016-11-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
WO2012117091A1 (en) 2011-03-02 2012-09-07 Novo Nordisk A/S Coagulation factor-targeting to tlt-1 on activated platelets
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.
WO2014070349A1 (en) * 2012-10-29 2014-05-08 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
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
ES2680932T3 (es) 2013-05-14 2018-09-11 Struszym, S.L. Métodos de determinación de actividades de los factores de coagulación
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
US11008561B2 (en) * 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US11066481B2 (en) 2015-07-23 2021-07-20 The Regents Of The University Of California Antibodies to coagulation factor XIa and uses thereof
CA3012695A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
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
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
AU2020242945B2 (en) 2019-03-19 2025-06-26 CSL Innovation Pty Ltd 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
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
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
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
US20250276092A1 (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

Family Cites Families (12)

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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
JP2004505016A (ja) 2000-05-10 2004-02-19 ノボ　ノルディスク　アクティーゼルスカブ 第ＶＩＩａ因子及び第ＸＩＩＩ因子を含んで成る医薬組成物
MXPA03001984A (es) * 2000-09-13 2003-08-29 Novo Nordisk Healthcare Ag Variantes de factor vii de coagulacion humano.
WO2002040544A2 (en) 2000-11-14 2002-05-23 Board Of Regents, University Of Texas Systems Mutant human factor ix with an increased resistance to inhibition by heparin
US20060040856A1 (en) * 2003-12-03 2006-02-23 Neose Technologies, Inc. Glycopegylated factor IX
WO2006018204A1 (en) * 2004-08-17 2006-02-23 Zlb Behring Gmbh Modified vitamin k dependent polypeptides
EP2423307A1 (en) * 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IV polypeptides and use thereof for treatment
US7700734B2 (en) * 2007-01-09 2010-04-20 Shu-Wha Lin Recombinant human factor IX and use thereof
AU2008209985A1 (en) * 2007-02-01 2008-08-07 Baxter Healthcare S.A. Improved fix-mutant proteins for hemophilia B treatment
US8022187B2 (en) * 2007-02-01 2011-09-20 Baxter International Inc. FVIII-independent FIX-mutant proteins for hemophilia A treatment
DE102008034724A1 (de) * 2008-07-25 2010-01-28 Alexander Koslow Verkugelungs- / Zerkleinerungsmaschine

Also Published As

Publication number Publication date
KR101768896B1 (ko) 2017-08-17
ES2753638T3 (es) 2020-04-13
US20110217284A1 (en) 2011-09-08
ES2441167T3 (es) 2014-02-03
ES2628688T3 (es) 2017-08-03
EP2390323A3 (en) 2012-01-11
US20190017039A1 (en) 2019-01-17
KR101947424B1 (ko) 2019-02-13
WO2010012451A1 (en) 2010-02-04
EP2767587A3 (en) 2014-11-26
EP2767587A2 (en) 2014-08-20
EP3623470A2 (en) 2020-03-18
EP2149603A1 (en) 2010-02-03
AU2009275391A1 (en) 2010-02-04
CA3033578C (en) 2022-07-26
AU2009275391B2 (en) 2015-01-22
CA2729501C (en) 2019-04-02
EP3241899A1 (en) 2017-11-08
CA2729501A1 (en) 2010-02-04
KR20110036071A (ko) 2011-04-06
EP3241899B1 (en) 2019-08-21
EP2318526A1 (en) 2011-05-11
CA3033578A1 (en) 2010-02-04
EP2390323B1 (en) 2013-11-27
EP2390323A2 (en) 2011-11-30
US10125357B2 (en) 2018-11-13
KR20170096210A (ko) 2017-08-23
EP2767587B1 (en) 2017-04-05
JP2011528913A (ja) 2011-12-01
CN102112608A (zh) 2011-06-29
EP3623470A3 (en) 2020-07-22
US10883097B2 (en) 2021-01-05
CN102112608B (zh) 2013-11-20

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