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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factor
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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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  • Urology & Nephrology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (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

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

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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) ES2753638T3 (enExample)
WO (1) WO2010012451A1 (enExample)

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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
RU2013142015A (ru) 2011-03-02 2015-04-10 Ново Нордиск А/С Нацеливание факторов свертывания крови на рецептор tlt-1 на поверхности активированных тромбоцитов
EP2737311B1 (en) 2011-07-25 2020-12-02 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
ES2959747T3 (es) 2013-02-15 2024-02-28 Bioverativ Therapeutics Inc Gen del factor VIII optimizado
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
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma 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
EP3411478B1 (en) 2016-02-01 2022-06-08 Bioverativ Therapeutics Inc. Optimized factor viii genes
EP4488288A3 (en) 2017-07-10 2025-04-16 uniQure IP B.V. Means and methods for aav gene therapy in humans
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
EP3941509B1 (en) * 2019-03-19 2025-12-03 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
US12403164B2 (en) 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
JP2023537565A (ja) 2020-06-24 2023-09-04 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 タンパク質を発現するように改変されたレンチウイルスベクターの調製物から遊離第ｖｉｉｉ因子を除去する方法
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
MX2024005124A (es) 2021-10-27 2024-05-16 Regeneron Pharma Composiciones y metodos para expresar el factor ix para terapia para la hemofilia b.
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

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CN1630721A (zh) * 2000-09-13 2005-06-22 诺沃挪第克健康护理股份公司 人凝血因子vii变体
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EP3241899B1 (en) 2019-08-21
CA2729501C (en) 2019-04-02
EP2149603A1 (en) 2010-02-03
ES2753638T3 (es) 2020-04-13
EP2390323B1 (en) 2013-11-27
WO2010012451A1 (en) 2010-02-04
EP2318526A1 (en) 2011-05-11
ES2441167T3 (es) 2014-02-03
EP3241899A1 (en) 2017-11-08
JP2011528913A (ja) 2011-12-01
CA2729501A1 (en) 2010-02-04
EP2390323A2 (en) 2011-11-30
KR101768896B1 (ko) 2017-08-17
EP2390323A3 (en) 2012-01-11
EP2767587B1 (en) 2017-04-05
EP2767587A2 (en) 2014-08-20
ES2628688T3 (es) 2017-08-03
KR101947424B1 (ko) 2019-02-13
US10883097B2 (en) 2021-01-05
EP3623470A2 (en) 2020-03-18
US20190017039A1 (en) 2019-01-17
CA3033578A1 (en) 2010-02-04
CA3033578C (en) 2022-07-26
AU2009275391B2 (en) 2015-01-22
CN102112608A (zh) 2011-06-29
KR20170096210A (ko) 2017-08-23
EP3623470A3 (en) 2020-07-22
EP2767587A3 (en) 2014-11-26
AU2009275391A1 (en) 2010-02-04
US10125357B2 (en) 2018-11-13
US20110217284A1 (en) 2011-09-08
JP6029826B2 (ja) 2016-11-24
KR20110036071A (ko) 2011-04-06

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