CA3033578C - Factor ix variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders - Google Patents

Factor ix variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders Download PDF

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Publication number
CA3033578C
CA3033578C CA3033578A CA3033578A CA3033578C CA 3033578 C CA3033578 C CA 3033578C CA 3033578 A CA3033578 A CA 3033578A CA 3033578 A CA3033578 A CA 3033578A CA 3033578 C CA3033578 C CA 3033578C
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Canada
Prior art keywords
variant
viii
activity
factor
amino acid
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Active
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CA3033578A
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English (en)
French (fr)
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CA3033578A1 (en
Inventor
Jorg Schuttrumpf
Erhard Seifried
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
DRK-Blutspendedienst Baden-Württemberg - Hessen
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DRK-Blutspendedienst Baden-Württemberg - Hessen
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Publication of CA3033578A1 publication Critical patent/CA3033578A1/en
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Classifications

    • 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
    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (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)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3033578A 2008-07-28 2009-07-28 Factor ix variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders Active CA3033578C (en)

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
CA2729501A CA2729501C (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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2729501A Division CA2729501C (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
CA3033578A1 CA3033578A1 (en) 2010-02-04
CA3033578C true CA3033578C (en) 2022-07-26

Family

ID=40032573

Family Applications (2)

Application Number Title Priority Date Filing Date
CA3033578A Active CA3033578C (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
CA2729501A Active CA2729501C (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

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2729501A Active CA2729501C (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

Country Status (9)

Country Link
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) CA3033578C (enExample)
ES (3) ES2628688T3 (enExample)
WO (1) WO2010012451A1 (enExample)

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ES2985035T3 (es) 2008-09-15 2024-11-04 Uniqure Biopharma B V Mutante de polipéptido de factor IX, sus usos y su procedimiento de producción
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
EA028914B1 (ru) * 2011-07-25 2018-01-31 Байоджен Хемофилия Инк. Исследования для мониторинга нарушений свертываемости крови
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
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
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
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
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
US11452749B2 (en) 2017-07-10 2022-09-27 Uniqure Ip B.V. Means and methods for AAV gene therapy in humans
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
ES3062513T3 (en) * 2019-03-19 2026-04-13 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
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
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
KR20240099358A (ko) 2021-10-27 2024-06-28 리제너론 파마슈티칼스 인코포레이티드 B형 혈우병 치료를 위한 인자 ix를 발현하기 위한 조성물 및 방법
CN116590262A (zh) * 2023-03-23 2023-08-15 上海交通大学医学院附属瑞金医院 一种凝血因子ix突变体及其应用
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
WO1999003496A1 (en) * 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
DK1282438T3 (da) 2000-05-10 2005-11-14 Novo Nordisk Healthcare Ag Farmaceutisk sammensætning omfattende en faktor VIIa og en faktor XIII
RU2326126C2 (ru) * 2000-09-13 2008-06-10 Ново Нордиск Хелт Кэр Аг Полипептид фактора vii свертывания крови, его получение и применение
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
RU2396347C2 (ru) * 2004-08-17 2010-08-10 Ксл Бехринг Гмбх Модифицированные витамин к-зависимые полипептиды
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
US8022187B2 (en) * 2007-02-01 2011-09-20 Baxter International Inc. FVIII-independent FIX-mutant proteins for hemophilia A treatment
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Also Published As

Publication number Publication date
AU2009275391B2 (en) 2015-01-22
EP2390323B1 (en) 2013-11-27
EP2767587A3 (en) 2014-11-26
KR101768896B1 (ko) 2017-08-17
ES2628688T3 (es) 2017-08-03
ES2441167T3 (es) 2014-02-03
EP3241899B1 (en) 2019-08-21
EP3623470A3 (en) 2020-07-22
JP2011528913A (ja) 2011-12-01
US20110217284A1 (en) 2011-09-08
EP2390323A3 (en) 2012-01-11
ES2753638T3 (es) 2020-04-13
WO2010012451A1 (en) 2010-02-04
KR20110036071A (ko) 2011-04-06
EP2390323A2 (en) 2011-11-30
JP6029826B2 (ja) 2016-11-24
AU2009275391A1 (en) 2010-02-04
KR20170096210A (ko) 2017-08-23
US10125357B2 (en) 2018-11-13
CA2729501A1 (en) 2010-02-04
CN102112608A (zh) 2011-06-29
EP2318526A1 (en) 2011-05-11
KR101947424B1 (ko) 2019-02-13
EP2767587A2 (en) 2014-08-20
CN102112608B (zh) 2013-11-20
CA2729501C (en) 2019-04-02
CA3033578A1 (en) 2010-02-04
EP2149603A1 (en) 2010-02-03
EP3241899A1 (en) 2017-11-08
EP2767587B1 (en) 2017-04-05
EP3623470A2 (en) 2020-03-18
US20190017039A1 (en) 2019-01-17
US10883097B2 (en) 2021-01-05

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Effective date: 20190212