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

Info

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
Authority
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
Other languages
English (en)
French (fr)
Other versions
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
Priority date (The priority date 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 date listed.)
Filing date
Publication date
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
Publication of CA2729501C publication Critical patent/CA2729501C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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

Landscapes

  • 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)
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
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

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) KR101947424B1 (https=)
CN (1) CN102112608B (https=)
AU (1) AU2009275391B2 (https=)
CA (2) CA2729501C (https=)
ES (3) ES2628688T3 (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
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)

* 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
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
JP6029826B2 (ja) 2016-11-24
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
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

Similar Documents

Publication Publication Date Title
US10883097B2 (en) Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders
US11208645B2 (en) 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
US9249404B2 (en) Coagulation factor X polypeptides with modified activation properties
JP6514893B2 (ja) 止血を調節するための組成物および方法
CN101356192B (zh) 用于调节止血的组合物和方法
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
WO2003084466A2 (en) FACTORVIIa COMPOSITIONS

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20140716

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 15TH ANNIV.) - STANDARD

Year of fee payment: 15

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240724

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240724

H13 Ip right lapsed

Free format text: ST27 STATUS EVENT CODE: N-4-6-H10-H13-H100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE AND LATE FEE NOT PAID BY DEADLINE OF NOTICE

Effective date: 20260323