WO2009140015A3 - Modification de facteur ix orientée site - Google Patents

Modification de facteur ix orientée site 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
Application number
PCT/US2009/040691
Other languages
English (en)
Other versions
WO2009140015A2 (fr
Inventor
Alan R. Brooks
John E. Murphy
Marian Seto
Xiaoqiao Jiang
David Kiewlich
Chandra Patel
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 JP2011505176A priority Critical patent/JP2011517950A/ja
Priority to CN2009801227857A priority patent/CN102065887A/zh
Priority to EP09747101A priority patent/EP2282767A4/fr
Priority to CA2721362A priority patent/CA2721362A1/fr
Publication of WO2009140015A2 publication Critical patent/WO2009140015A2/fr
Publication of WO2009140015A3 publication Critical patent/WO2009140015A3/fr

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

Abstract

L'invention concerne des polypeptides du facteur IX modifié, tels que des polypeptides du facteur IX dans lesquels un ou plusieurs sites cystéine ont été introduits. Les polypeptides du facteur IX modifié peuvent être conjugués avec un polymère biocompatible. L'invention concerne également des procédés pour produire des polypeptides du facteur IX modifié, et des méthodes pour utiliser lesdits polypeptides du facteur IX modifié, par exemple, pour traiter des patients souffrant d'hémophilie B.
PCT/US2009/040691 2008-04-16 2009-04-15 Modification de facteur ix orientée site WO2009140015A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2011505176A JP2011517950A (ja) 2008-04-16 2009-04-15 第ｉｘ因子の部位特異的修飾
CN2009801227857A CN102065887A (zh) 2008-04-16 2009-04-15 因子ix的定点修饰
EP09747101A EP2282767A4 (fr) 2008-04-16 2009-04-15 Modification de facteur ix orientée site
CA2721362A CA2721362A1 (fr) 2008-04-16 2009-04-15 Modification de facteur ix orientee site

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 (fr) 2009-11-19
WO2009140015A3 true WO2009140015A3 (fr) 2010-01-07

Family

ID=41319239

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/040691 WO2009140015A2 (fr) 2008-04-16 2009-04-15 Modification de facteur ix orientée site

Country Status (6)

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

Families Citing this family (36)

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Publication number Priority date Publication date Assignee Title
PL2337849T3 (pl) 2008-09-15 2018-11-30 Uniqure Biopharma B.V. Zmutowany polipeptyd czynnika ix, jego zastosowania i sposób jego otrzymywania
EP3552619A1 (fr) 2010-07-09 2019-10-16 Bioverativ Therapeutics Inc. Polypeptides du facteur ix et leurs procédés d'utilisation
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
PT2717898T (pt) 2011-06-10 2019-05-20 Bioverativ Therapeutics Inc Compostos pró-coagulantes e processos para a sua utilização
WO2013016454A1 (fr) 2011-07-25 2013-01-31 Biogen Idec Hemophilia Inc. Dosages pour la surveillance de troubles hémostatiques
EA033469B1 (ru) * 2012-04-16 2019-10-31 Cantab Biopharmaceuticals Patents Ltd Подкожное введение конъюгатов факторов крови с полиэтиленгликолем
WO2013162078A1 (fr) 2012-04-27 2013-10-31 学校法人日本大学 Agent thérapeutique pour une lésion dans un épithélium et un endothélium
EP2877202A4 (fr) 2012-07-25 2016-06-01 Biogen Ma Inc Essai de surveillance de facteur sanguin et utilisations de celui-ci
EP3542861A1 (fr) 2012-09-25 2019-09-25 Bioverativ Therapeutics Inc. Procédés d'utilisation de polypeptides fix
WO2014063108A1 (fr) 2012-10-18 2014-04-24 Biogen Idec Ma Inc. Procédés d'utilisation d'une dose fixe d'un facteur de coagulation
US10717965B2 (en) 2013-01-10 2020-07-21 Gloriana Therapeutics, Inc. Mammalian cell culture-produced neublastin antibodies
US10370431B2 (en) 2013-02-15 2019-08-06 Bioverativ Therapeutics Inc. Optimized factor VIII gene
SG11201505926VA (en) 2013-03-15 2015-09-29 Biogen Ma Inc Factor ix polypeptide formulations
US10611794B2 (en) 2013-09-25 2020-04-07 Bioverativ Therapeutics Inc. On-column viral inactivation methods
EP3065769A4 (fr) 2013-11-08 2017-05-31 Biogen MA Inc. Composé de fusion procoagulant
EP4332839A2 (fr) 2013-12-06 2024-03-06 Bioverativ Therapeutics Inc. Outils pharmacocinétiques de population et leurs utilisations
WO2015095809A1 (fr) 2013-12-20 2015-06-25 Biogen Idec Ma Inc. Utilisation de cultures de semences sous perfusion pour améliorer la capacité de production en alimentation programmée de produits biopharmaceutiques et la qualité des produits
US10772942B2 (en) 2014-03-24 2020-09-15 Bioverativ Therapeutics Inc. Lyophilized factor IX formulations
WO2016004113A1 (fr) 2014-06-30 2016-01-07 Biogen Ma Inc. Gène du facteur ix optimisé
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
TWI741992B (zh) 2015-08-03 2021-10-11 美商百歐維拉提夫治療公司 因子ｉｘ融合蛋白以及其製備及使用方法
PT3411478T (pt) 2016-02-01 2022-09-13 Bioverativ Therapeutics Inc Genes do fator viii otimizados
US20190381149A1 (en) 2016-12-02 2019-12-19 Bioverativ Therapeutics Inc. Methods of treating hemophilic arthropathy using chimeric clotting factors
MX2019009063A (es) 2017-01-31 2019-10-21 Bioverativ Therapeutics Inc Proteinas de fusion del factor ix y procedimientos de preparacion y utilizacion de las mismas.
CN111247251A (zh) 2017-08-09 2020-06-05 比奥维拉迪维治疗股份有限公司 核酸分子及其用途
US11491212B1 (en) 2017-09-27 2022-11-08 Catalyst Biosciences, Inc. Subcutaneous administration of modified factor IX polypeptides and treatment of hemophilia B
AU2018338608A1 (en) 2017-09-27 2020-04-09 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
CA3096038A1 (fr) 2018-04-04 2019-10-10 Sigilon Therapeutics, Inc. Particules implantables et procedes associes
US20210145889A1 (en) 2018-04-04 2021-05-20 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising stem cells
SG11202101157VA (en) 2018-08-09 2021-03-30 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof for non-viral gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
UY38389A (es) 2018-09-27 2020-04-30 Sigilon Therapeutics Inc Dispositivos implantables para terapia celular y métodos relacionados
US20220323519A1 (en) 2019-04-17 2022-10-13 Codiak Biosciences, Inc. Compositions of exosomes and aav
WO2021154414A2 (fr) 2020-01-29 2021-08-05 Catalyst Biosciences, Inc. Thérapie génique pour l'hémophilie b avec un vecteur de capside d'aav chimérique codant pour des polypeptides du facteur ix modifiés
WO2024081309A1 (fr) 2022-10-11 2024-04-18 Sigilon Therapeutics, Inc. Cellules modifiées et éléments implantables pour le traitement d'une maladie
WO2024081310A1 (fr) 2022-10-11 2024-04-18 Sigilon Therapeutics, Inc. Cellules modifiées et éléments implantables pour le traitement d'une maladie

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 (fr) * 2004-06-30 2006-01-12 Nektar Therapeutics Al, Corporation Conjugues polymere-fraction facteur ix
WO2007149406A2 (fr) * 2006-06-19 2007-12-27 Nautilus Technology Llc Polypeptides facteurs ix de la coagulation modifiés et leur utilisation thérapeutique

Family Cites Families (1)

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Publication number Priority date Publication date Assignee Title
RU2008145084A (ru) * 2006-05-24 2010-06-27 Ново Нордиск Хелс Кеа Аг (Ch) Аналоги фактора ix, имеющие пролонгированное время полужизни in vivo

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 (fr) * 2004-06-30 2006-01-12 Nektar Therapeutics Al, Corporation Conjugues polymere-fraction facteur ix
WO2007149406A2 (fr) * 2006-06-19 2007-12-27 Nautilus Technology Llc Polypeptides facteurs ix de la coagulation modifiés et leur utilisation thérapeutique

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
CN102065887A (zh) 2011-05-18
WO2009140015A2 (fr) 2009-11-19
CA2721362A1 (fr) 2009-11-19
EP2282767A2 (fr) 2011-02-16
JP2011517950A (ja) 2011-06-23
EP2282767A4 (fr) 2012-07-11
KR20110015551A (ko) 2011-02-16

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