CA2729501C - 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
CA2729501C
CA2729501C CA2729501A CA2729501A CA2729501C CA 2729501 C CA2729501 C CA 2729501C CA 2729501 A CA2729501 A CA 2729501A CA 2729501 A CA2729501 A CA 2729501A CA 2729501 C CA2729501 C CA 2729501C
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CA
Canada
Prior art keywords
variant
factor
viii
activity
amino acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CA2729501A
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English (en)
French (fr)
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CA2729501A1 (en
Inventor
Erhard Seifried
Joerg Schuettrumpf
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 Wuerttermberg Hessen gGmbH
Original Assignee
DRK Blutspendedienst Baden Wuerttermberg Hessen gGmbH
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Application filed by DRK Blutspendedienst Baden Wuerttermberg Hessen gGmbH filed Critical DRK Blutspendedienst Baden Wuerttermberg Hessen gGmbH
Priority to CA3033578A priority Critical patent/CA3033578C/en
Publication of CA2729501A1 publication Critical patent/CA2729501A1/en
Application granted granted Critical
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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
    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • 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)
CA2729501A 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 CA2729501C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3033578A 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

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3033578A Division 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

Publications (2)

Publication Number Publication Date
CA2729501A1 CA2729501A1 (en) 2010-02-04
CA2729501C true CA2729501C (en) 2019-04-02

Family

ID=40032573

Family Applications (2)

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
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

Family Applications After (1)

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

Country Status (9)

Country Link
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=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4219547A3 (en) 2008-09-15 2023-10-18 uniQure biopharma B.V. Factor ix polypeptide mutant, its uses and a method for its production
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
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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CA2406583A1 (en) 2000-05-10 2001-11-15 Novo Nordisk A/S Pharmaceutical composition comprising a factor viia and a factor xiii
MXPA03001984A (es) * 2000-09-13 2003-08-29 Novo Nordisk Healthcare Ag Variantes de factor vii de coagulacion humano.
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
US20060040856A1 (en) * 2003-12-03 2006-02-23 Neose Technologies, Inc. Glycopegylated factor IX
ATE469216T1 (de) * 2004-08-17 2010-06-15 Csl Behring Gmbh Modifizierte vitamin-k-abhängige polypeptide
EP2423305A1 (en) * 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IX polypeptides and use thereof for treatment
US7700734B2 (en) * 2007-01-09 2010-04-20 Shu-Wha Lin Recombinant human factor IX and use thereof
WO2008092643A2 (en) * 2007-02-01 2008-08-07 Baxter International Inc. Improved fix-mutant proteins for hemophilia b treatment
CA2674881A1 (en) * 2007-02-01 2008-08-07 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
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
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
CN102112608B (zh) 2013-11-20

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Class et al. Patent application title: 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
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