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WO2009140015A3 - Site-directed modification of factor ix - Google Patents

Site-directed modification of factor ix Download PDF

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Publication number
WO2009140015A3
WO2009140015A3 PCT/US2009/040691 US2009040691W WO2009140015A3 WO 2009140015 A3 WO2009140015 A3 WO 2009140015A3 US 2009040691 W US2009040691 W US 2009040691W WO 2009140015 A3 WO2009140015 A3 WO 2009140015A3
Authority
WO
WIPO (PCT)
Prior art keywords
factor
polypeptides
site
directed modification
modified factor
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.)
Ceased
Application number
PCT/US2009/040691
Other languages
French (fr)
Other versions
WO2009140015A2 (en
Inventor
Alan R. Brooks
John E. Murphy
Marian Seto
Xiaoqiao Jiang
David Kiewlich
Chandra Patel
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.)
Bayer Healthcare LLC
Original Assignee
Bayer Healthcare LLC
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 Bayer Healthcare LLC filed Critical Bayer Healthcare LLC
Priority to EP09747101A priority Critical patent/EP2282767A4/en
Priority to CN2009801227857A priority patent/CN102065887A/en
Priority to CA2721362A priority patent/CA2721362A1/en
Priority to JP2011505176A priority patent/JP2011517950A/en
Publication of WO2009140015A2 publication Critical patent/WO2009140015A2/en
Publication of WO2009140015A3 publication Critical patent/WO2009140015A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The invention relates to modified Factor IX polypeptides such as Factor IX polypeptides with one or more introduced cysteine sites. The modified Factor IX polypeptides may be conjugated to a biocompatible polymer. The invention also relates to methods of making modified Factor IX polypeptides, and methods of using modified Factor IX polypeptides, for example, to treat patients afflicted with hemophilia B.
PCT/US2009/040691 2008-04-16 2009-04-15 Site-directed modification of factor ix Ceased WO2009140015A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP09747101A EP2282767A4 (en) 2008-04-16 2009-04-15 Site-directed modification of factor ix
CN2009801227857A CN102065887A (en) 2008-04-16 2009-04-15 Site-directed modification of factor IX
CA2721362A CA2721362A1 (en) 2008-04-16 2009-04-15 Site-directed modification of factor ix
JP2011505176A JP2011517950A (en) 2008-04-16 2009-04-15 Site-specific modification of factor IX

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US12456808P 2008-04-16 2008-04-16
US61/124,568 2008-04-16

Publications (2)

Publication Number Publication Date
WO2009140015A2 WO2009140015A2 (en) 2009-11-19
WO2009140015A3 true WO2009140015A3 (en) 2010-01-07

Family

ID=41319239

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/040691 Ceased WO2009140015A2 (en) 2008-04-16 2009-04-15 Site-directed modification of factor ix

Country Status (6)

Country Link
EP (1) EP2282767A4 (en)
JP (1) JP2011517950A (en)
KR (1) KR20110015551A (en)
CN (1) CN102065887A (en)
CA (1) CA2721362A1 (en)
WO (1) WO2009140015A2 (en)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2737094C (en) 2008-09-15 2018-02-20 Paolo Simioni Factor ix polypeptide mutant, its uses and a method for its production
SG186856A1 (en) 2010-07-09 2013-02-28 Biogen Idec Hemophilia Inc Factor ix polypeptides and methods of use thereof
TWI557135B (en) 2010-11-03 2016-11-11 介控生化科技公司 Modified ninth factor multi-peptide and use thereof
PL2717898T3 (en) 2011-06-10 2019-06-28 Bioverativ Therapeutics Inc. Pro-coagulant compounds and methods of use thereof
US10656167B2 (en) 2011-07-25 2020-05-19 Bioverativ Therapeutics Inc. Assays to monitor bleeding disorders
EP2838566A2 (en) 2012-04-16 2015-02-25 Cantab Biopharmaceuticals Patents Limited Optimised subcutaneous therapeutic agents
US9738884B2 (en) 2012-04-27 2017-08-22 Nihon University Therapeutic agent for epithelial and endothelial injury
HK1212882A1 (en) 2012-07-25 2016-06-24 Bioverativ Therapeutics Inc. Blood factor monitoring assay and uses thereof
EP2900328A4 (en) 2012-09-25 2016-05-11 Biogen Ma Inc Methods of using fix polypeptides
US10391152B2 (en) 2012-10-18 2019-08-27 Bioverativ Therapeutics Inc. Methods of using a fixed dose of a clotting factor
US10717965B2 (en) 2013-01-10 2020-07-21 Gloriana Therapeutics, Inc. Mammalian cell culture-produced neublastin antibodies
DK3889173T5 (en) 2013-02-15 2024-08-05 Bioverativ Therapeutics Inc OPTIMIZED FACTOR VIII GENE
WO2014144549A1 (en) 2013-03-15 2014-09-18 Biogen Idec Ma Inc. Factor ix polypeptide formulations
HUE057005T2 (en) 2013-09-25 2022-04-28 Bioverativ Therapeutics Inc Column virus inactivation procedures
WO2015070014A1 (en) 2013-11-08 2015-05-14 Biogen Idec Ma Inc. Procoagulant fusion compound
ES2967617T3 (en) 2013-12-06 2024-05-03 Bioverativ Therapeutics Inc Population pharmacokinetics tools and their uses
JP2017500017A (en) 2013-12-20 2017-01-05 バイオジェン・エムエイ・インコーポレイテッドＢｉｏｇｅｎ ＭＡ Ｉｎｃ． Use of perfusion seed cultures to improve biopharmaceutical fed-batch production capacity and product quality
JP7058940B2 (en) 2014-03-24 2022-04-25 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 Freeze-dried factor IX preparation
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
EA201890423A1 (en) 2015-08-03 2018-07-31 Биовератив Терапьютикс Инк. SLIGHT PROTEINS OF THE FACTOR IX, METHODS OF THEIR RECEPTION AND APPLICATION
AU2017214378B2 (en) 2016-02-01 2023-05-04 Bioverativ Therapeutics Inc. Optimized Factor VIII genes
JP7748175B2 (en) 2016-12-02 2025-10-02 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 Methods for treating hemophilic arthropathy using chimeric clotting factors - Patent Application 20070122999
BR112019015569A2 (en) 2017-01-31 2020-03-17 Bioverativ Therapeutics Inc. FACTOR IX FUSION PROTEINS AND METHODS FOR THEIR PRODUCTION AND USE
BR112020002394A2 (en) 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. nucleic acid molecules and uses thereof
MX2020003351A (en) 2017-09-27 2020-10-12 Sigilon Therapeutics Inc Methods, compositions, and implantable elements comprising active cells.
US11491212B1 (en) 2017-09-27 2022-11-08 Catalyst Biosciences, Inc. Subcutaneous administration of modified factor IX polypeptides and treatment of hemophilia B
US20210145889A1 (en) 2018-04-04 2021-05-20 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising stem cells
BR112020020084A2 (en) 2018-04-04 2021-01-05 Sigilon Therapeutics, Inc. PARTICLE, PREPARATION OF A PARTICLE PLURALITY, METHOD OF PREPARATION OF A PARTICLE, AND, PARTICLE COMPOSITION.
CN112512555A (en) 2018-05-18 2021-03-16 比奥维拉迪维治疗股份有限公司 How to treat hemophilia A
JP7602454B2 (en) 2018-08-09 2024-12-18 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 Nucleic Acid Molecules and Their Use for Non-Viral Gene Therapy - Patent application
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
UY38389A (en) 2018-09-27 2020-04-30 Sigilon Therapeutics Inc IMPLANTABLE DEVICES FOR CELLULAR THERAPY AND RELATED METHODS
WO2020215010A1 (en) 2019-04-17 2020-10-22 Codiak Biosciences, Inc. Compositions of exosomes and aav
TW202126284A (en) 2019-09-30 2021-07-16 美商百歐維拉提夫治療公司 Lentiviral vector formulations
WO2021154414A2 (en) 2020-01-29 2021-08-05 Catalyst Biosciences, Inc. Gene therapy for hemophilia b with a chimeric aav capsid vector encoding modified factor ix polypeptides
KR20230074703A (en) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. Method for removing free factor VIII from preparations of lentiviral vectors modified to express the protein
WO2024081310A1 (en) 2022-10-11 2024-04-18 Sigilon Therapeutics, Inc. Engineered cells and implantable elements for treatment of disease
WO2024081309A1 (en) 2022-10-11 2024-04-18 Sigilon Therapeutics, Inc. Engineered cells and implantable elements for treatment of disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6531298B2 (en) * 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
WO2006005058A2 (en) * 2004-06-30 2006-01-12 Nektar Therapeutics Al, Corporation Polymer-factor ix moiety conjugates
WO2007149406A2 (en) * 2006-06-19 2007-12-27 Nautilus Technology Llc Modified coagulation factor ix polypeptides and use thereof for treatment

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2029738A2 (en) * 2006-05-24 2009-03-04 Novo Nordisk Health Care AG Factor ix analogues having prolonged in vivo half life

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6531298B2 (en) * 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
WO2006005058A2 (en) * 2004-06-30 2006-01-12 Nektar Therapeutics Al, Corporation Polymer-factor ix moiety conjugates
WO2007149406A2 (en) * 2006-06-19 2007-12-27 Nautilus Technology Llc Modified coagulation factor ix polypeptides and use thereof for treatment

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
MCGRAW ET AL.: "Evidence for a prevalent dimorphism in the activation peptide of human coagulation factor IX.", PROC NAT ACAD SCI, vol. 82, no. 9, May 1985 (1985-05-01), pages 2847 - 2851, XP008146066 *
SARKAR ET AL.: "Direct sequencing of the activation peptide and the catalytic domain of the factor IX gene in six species.", GENOMICS, vol. 6, no. 1, January 1990 (1990-01-01), pages 133 - 143, XP024797431 *
See also references of EP2282767A4 *

Also Published As

Publication number Publication date
EP2282767A2 (en) 2011-02-16
CA2721362A1 (en) 2009-11-19
KR20110015551A (en) 2011-02-16
WO2009140015A2 (en) 2009-11-19
CN102065887A (en) 2011-05-18
EP2282767A4 (en) 2012-07-11
JP2011517950A (en) 2011-06-23

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