ATE209679T1 - Proteine mit faktor viii aktivität, verfahren zu deren herstellung unter verwendung von genetisch modifizierten zellen und diese enthaltende zusammensetzungen - Google Patents

Proteine mit faktor viii aktivität, verfahren zu deren herstellung unter verwendung von genetisch modifizierten zellen und diese enthaltende zusammensetzungen

Info

Publication number
ATE209679T1
ATE209679T1 AT95110468T AT95110468T ATE209679T1 AT E209679 T1 ATE209679 T1 AT E209679T1 AT 95110468 T AT95110468 T AT 95110468T AT 95110468 T AT95110468 T AT 95110468T AT E209679 T1 ATE209679 T1 AT E209679T1
Authority
AT
Austria
Prior art keywords
factor viii
proteins
production
compositions containing
genetically modified
Prior art date
Application number
AT95110468T
Other languages
English (en)
Inventor
Ooyen Albert Johannes Jose Van
Hans Pannekoek
Martinus P Verbeet
Leen Robert Willem Van
Original Assignee
Baxter Ag
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 Baxter Ag filed Critical Baxter Ag
Application granted granted Critical
Publication of ATE209679T1 publication Critical patent/ATE209679T1/de

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/36—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against blood coagulation factors
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S530/00—Chemistry: natural resins or derivatives; peptides or proteins; lignins or reaction products thereof
    • Y10S530/827—Proteins from mammals or birds
    • Y10S530/829—Blood
    • Y10S530/83—Plasma; serum

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AT95110468T 1987-06-12 1988-06-13 Proteine mit faktor viii aktivität, verfahren zu deren herstellung unter verwendung von genetisch modifizierten zellen und diese enthaltende zusammensetzungen ATE209679T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP87201121 1987-06-12

Publications (1)

Publication Number Publication Date
ATE209679T1 true ATE209679T1 (de) 2001-12-15

Family

ID=8197627

Family Applications (2)

Application Number Title Priority Date Filing Date
AT95110468T ATE209679T1 (de) 1987-06-12 1988-06-13 Proteine mit faktor viii aktivität, verfahren zu deren herstellung unter verwendung von genetisch modifizierten zellen und diese enthaltende zusammensetzungen
AT88201209T ATE142691T1 (de) 1987-06-12 1988-06-13 Proteine mit faktor-viii-aktivität, verfahren zu deren herstellung unter verwendung von genetisch modifizierten zellen und diese enthaltende pharmazeutische zusammensetzungen

Family Applications After (1)

Application Number Title Priority Date Filing Date
AT88201209T ATE142691T1 (de) 1987-06-12 1988-06-13 Proteine mit faktor-viii-aktivität, verfahren zu deren herstellung unter verwendung von genetisch modifizierten zellen und diese enthaltende pharmazeutische zusammensetzungen

Country Status (19)

Country Link
US (2) US5171844A (de)
JP (1) JP2771827B2 (de)
KR (1) KR970002915B1 (de)
CN (1) CN1033760C (de)
AT (2) ATE209679T1 (de)
CA (1) CA1341208C (de)
DE (2) DE3855523T2 (de)
DK (1) DK172631B1 (de)
ES (2) ES2169095T3 (de)
FI (1) FI103731B (de)
GR (1) GR3021545T3 (de)
HU (2) HU213300B (de)
IE (1) IE69026B1 (de)
IL (1) IL86693A (de)
NO (1) NO301121B1 (de)
NZ (1) NZ224992A (de)
PT (1) PT87721B (de)
WO (1) WO1988009813A1 (de)
ZA (1) ZA884216B (de)

Families Citing this family (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5422260A (en) * 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US6060447A (en) * 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
JPH0387173A (ja) * 1987-09-10 1991-04-11 Teijin Ltd ヒト活性化天然型ファクター8cの製造方法及びそれに用いる形質転換体
FR2657884B1 (fr) * 1990-02-05 1994-09-02 Tm Innovation Procede pour la preparation du facteur viii humain et d'analogues du facteur viii.
EP0548214B1 (de) * 1990-09-14 1999-03-10 Chiron Corporation Expression von makrophagen-induzierbaren proteinen (mips) in hefezellen
US20060122376A1 (en) * 1991-02-07 2006-06-08 Chiron Corporation Protein complexes having factor VIII:C activity and production thereof
US5888974A (en) * 1992-04-07 1999-03-30 Emory University Hybrid human/animal factor VIII
US5859204A (en) * 1992-04-07 1999-01-12 Emory University Modified factor VIII
SE504074C2 (sv) * 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
US5576291A (en) * 1993-09-13 1996-11-19 Baxter International Inc. Activated factor VIII as a therapeutic agent and method of treating factor VIII deficiency
AT403167B (de) * 1994-11-14 1997-11-25 Immuno Ag Selektion und expression von fremdproteinen mittels eines selektions-amplifikations-systems
US5641655A (en) * 1994-11-30 1997-06-24 Zymogenetics, Inc. Methods for producing thrombopoietin polypeptides using a mammalian tissue plasminogen activator secretory peptide
AU5989796A (en) * 1995-06-06 1996-12-24 Board Of Regents Of The University Of Nebraska, by and on behalf of The University Of Nebraska Medical Center, The Organ function replacement compositions and methods of makin g and using the same
US7560107B2 (en) * 1996-06-26 2009-07-14 Emory University Modified factor VIII
JP2002517180A (ja) 1997-12-05 2002-06-18 ジ・イミユーン・リスポンス・コーポレーシヨン 増大された発現を示す新規ベクターおよび遺伝子
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6358703B1 (en) * 1998-12-10 2002-03-19 Bayer Corporation Expression system for factor VIII
EP1325681A1 (de) * 2001-12-11 2003-07-09 Société des Produits Nestlé S.A. Zusammensetzung für die Förderung des Knochenwachstums und die Erhaltung der Knochen
US7211558B2 (en) * 2002-05-22 2007-05-01 The Children's Hospital Of Philadelphia Compositions and methods for the treatment of hemophilia A
TWI353991B (en) 2003-05-06 2011-12-11 Syntonix Pharmaceuticals Inc Immunoglobulin chimeric monomer-dimer hybrids
EP1668143B1 (de) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Claden und sequenzen von adeno-assoziiertem virus (aav), diese enthaltende vektoren sowie verwendungen dafür
AU2005282380A1 (en) 2004-09-07 2006-03-16 Archemix Corp. Aptamer medicinal chemistry
US7566701B2 (en) 2004-09-07 2009-07-28 Archemix Corp. Aptamers to von Willebrand Factor and their use as thrombotic disease therapeutics
JP2008512098A (ja) 2004-09-07 2008-04-24 アーケミックス コーポレイション フォン・ビルブラント因子に対するアプタマー、および血栓性疾患の処置剤としてのその使用
DK2371856T3 (en) 2004-11-12 2022-08-08 Bayer Healthcare Llc Site-directed modifikation af FVIII
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
US7855279B2 (en) 2005-09-27 2010-12-21 Amunix Operating, Inc. Unstructured recombinant polymers and uses thereof
SI2574677T1 (sl) 2007-12-27 2017-11-30 Baxalta GmbH Procesi celične kulture
TWI492951B (zh) 2007-12-31 2015-07-21 Baxter Int 表現人類凝血因子的基因轉殖非人類動物及其用途
DK3178835T3 (da) 2009-02-03 2019-06-24 Amunix Pharmaceuticals Inc Forlængede rekombinante polypeptider og sammensætninger omfattende samme
US20110072524A1 (en) * 2009-08-04 2011-03-24 Baxter International Inc. Transgenic Mouse Lacking Endogenous FVIII and VWF - A Model of Hemophilia A
CA2771999A1 (en) 2009-08-24 2011-03-10 Amunix Operating Inc. Coagulation factor vii compositions and methods of making and using same
EP4382170A3 (de) 2009-12-06 2024-09-04 Bioverativ Therapeutics Inc. Chimäre und hybride faktor-viii-fc-polypeptide und verfahren zur verwendung davon
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9611310B2 (en) 2010-07-09 2017-04-04 Bioverativ Therapeutics Inc. Systems for factor VIII processing and methods thereof
PT2591006T (pt) 2010-07-09 2019-07-29 Bioverativ Therapeutics Inc Moléculas de cadeia simples processáveis e polipéptidos preparados utilizando as mesmas
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
CA2838833A1 (en) 2011-06-10 2012-12-13 Biogen Idec Ma Inc. Pro-coagulant compounds and methods of use thereof
EP4169525A1 (de) 2011-07-08 2023-04-26 Bioverativ Therapeutics Inc. Chimäre und hybride faktor-viii-polypeptide und verfahren zur verwendung davon
EA028914B1 (ru) 2011-07-25 2018-01-31 Байоджен Хемофилия Инк. Исследования для мониторинга нарушений свертываемости крови
CN103917554B (zh) 2011-10-18 2017-03-08 杰特有限公司 用于改善重构后纯化的因子viii的稳定性的方法
EA038705B1 (ru) 2012-01-12 2021-10-07 Биовератив Терапьютикс Инк. Способы снижения иммуногенности фактора свёртывания крови viii у пациентов, получающих лечение фактором viii
HRP20191920T1 (hr) 2012-01-12 2020-01-10 Bioverativ Therapeutics Inc. Kimerni polipeptidi faktora viii i njihova uporaba
CN111548418A (zh) 2012-02-15 2020-08-18 比奥贝拉蒂治疗公司 因子viii组合物及其制备和使用方法
WO2013123457A1 (en) 2012-02-15 2013-08-22 Biogen Idec Ma Inc. Recombinant factor viii proteins
AU2013270682A1 (en) 2012-06-08 2014-12-11 Biogen Ma Inc. Procoagulant compounds
AU2013270683A1 (en) 2012-06-08 2014-12-11 Biogen Ma Inc. Chimeric clotting factors
US10023628B2 (en) 2012-07-06 2018-07-17 Bioverativ Therapeutics Inc. Cell line expressing single chain factor VIII polypeptides and uses thereof
ES2770501T3 (es) 2012-07-11 2020-07-01 Bioverativ Therapeutics Inc Complejo del factor VIII con XTEN y proteína del factor de Von Willebrand y sus usos
WO2014018777A2 (en) 2012-07-25 2014-01-30 Biogen Idec Ma Inc. Blood factor monitoring assay and uses thereof
AU2013331000B2 (en) 2012-10-18 2018-04-19 Bioverativ Therapeutics Inc. Methods of using a fixed dose of a clotting factor
WO2014070953A1 (en) 2012-10-30 2014-05-08 Biogen Idec Ma Inc. Methods of Using FVIII Polypeptide
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
SG11201505926VA (en) 2013-03-15 2015-09-29 Biogen Ma Inc Factor ix polypeptide formulations
ES2926798T3 (es) 2013-03-15 2022-10-28 Bioverativ Therapeutics Inc Formulaciones de polipéptido de factor VIII
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
KR20230007555A (ko) 2013-06-28 2023-01-12 바이오버라티브 테라퓨틱스 인크. Xten을 지닌 트롬빈 절단가능한 링커 및 이의 용도
EP3875106A1 (de) 2013-08-08 2021-09-08 Bioverativ Therapeutics Inc. Reinigung von chimären fviii-molekülen
US10548953B2 (en) 2013-08-14 2020-02-04 Bioverativ Therapeutics Inc. Factor VIII-XTEN fusions and uses thereof
WO2015070014A1 (en) 2013-11-08 2015-05-14 Biogen Idec Ma Inc. Procoagulant fusion compound
US10325687B2 (en) 2013-12-06 2019-06-18 Bioverativ Therapeutics Inc. Population pharmacokinetics tools and uses thereof
EA201691111A1 (ru) 2014-01-10 2017-01-30 Байоджен Ма Инк. Химерные белки фактора viii и их применение
NZ722391A (en) 2014-02-04 2022-12-23 Biogen Ma Inc Use of cation-exchange chromatography in the flow-through mode to enrich post-translational modifications
CN105017410B (zh) * 2014-04-18 2018-06-08 北京诺思兰德生物技术股份有限公司 一种b区部分缺失型重组人凝血因子ⅷ
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
MA40864A (fr) 2014-10-31 2017-09-05 Biogen Ma Inc Hypotaurine, gaba, bêta-alanine et choline pour la régulation de l'accumulation de sous-produits résiduaires dans des procédés de culture de cellules mammifères
CN115094062A (zh) 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
UA126016C2 (uk) 2015-08-03 2022-08-03 Біовератів Терапеутікс Інк. Злитий білок фактора іх
KR102404550B1 (ko) 2015-11-13 2022-05-31 다케다 야쿠힌 고교 가부시키가이샤 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
CN108884145B (zh) 2015-11-13 2023-08-22 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
JP6877469B2 (ja) 2016-06-24 2021-05-26 モガム・インスティテュート・フォー・バイオメディカル・リサーチMogam Institute For Biomedical Research Fviiiおよびvwf因子を含むキメラタンパク質、ならびにそれらの使用
KR20190090827A (ko) 2016-12-02 2019-08-02 바이오버라티브 테라퓨틱스 인크. 키메라 응고 인자를 사용해 혈우병성 관절증을 치료하는 방법
CA3044838A1 (en) 2016-12-02 2018-06-07 Bioverativ Therapeutics Inc. Methods of inducing immune tolerance to clotting factors
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
US20210038744A1 (en) 2018-02-01 2021-02-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor viii
CN112203646A (zh) 2018-04-04 2021-01-08 西吉隆医疗股份有限公司 可植入颗粒和相关方法
KR102914074B1 (ko) 2018-05-18 2026-01-16 바이오버라티브 테라퓨틱스 인크. A형 혈우병의 치료 방법
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
JP7602454B2 (ja) 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 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
UY38389A (es) 2018-09-27 2020-04-30 Sigilon Therapeutics Inc Dispositivos implantables para terapia celular y métodos relacionados
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
EP3736286A1 (de) 2019-05-09 2020-11-11 Biotest AG Einzelkettenfaktor-viii-molekül
AU2020298233A1 (en) 2019-06-19 2022-01-20 Bioverativ Therapeutics Inc. Recombinant factor VIII-Fc for treating hemophilia and low bone mineral density
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
CN114981299A (zh) 2019-12-12 2022-08-30 武田药品工业株式会社 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法
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EP4355768A1 (de) 2021-06-14 2024-04-24 Takeda Pharmaceutical Company Limited Gentherapie von hämophilie a unter verwendung von für rekombinante fviii-varianten codierenden viralen vektoren mit erhöhter expression
US20230084036A1 (en) 2021-08-23 2023-03-16 Bioverativ Therapeutics Inc. Optimized factor viii genes
KR20240067122A (ko) 2021-09-30 2024-05-16 바이오버라티브 테라퓨틱스 인크. 감소된 면역원성을 갖는 인자 viii 폴리펩타이드를 암호화하는 핵산
WO2024081309A1 (en) 2022-10-11 2024-04-18 Sigilon Therapeutics, Inc. Engineered cells and implantable elements for treatment of disease
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4657894A (en) 1983-03-31 1987-04-14 Scripps Clinic & Research Foundation New factor VIII coagulant polypeptides
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4857635A (en) 1983-03-31 1989-08-15 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides and monoclonal antibodies tof them
US5004804A (en) * 1984-01-12 1991-04-02 Nordisk Gentofte Method and composition for preparation of factor VIIIC
IL74909A (en) * 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
US4965199A (en) * 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
SE8501050D0 (sv) 1985-03-05 1985-03-05 Kabivitrum Ab Biologically active fragments of human antihemophilic factor and method for preparation thereof
MX9203440A (es) * 1985-04-12 1992-07-01 Genetics Inst Proteinas procoagulantes novedosas.
US4769336A (en) 1985-05-24 1988-09-06 Scripps Clinic And Research Foundation Treatment of factor VIII inhibitors
KR910006424B1 (ko) * 1985-08-21 1991-08-24 인코텍스 비.브이 편성브리프(brief) 제조방법
FI98829C (fi) * 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5451521A (en) * 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
EP0251843A1 (de) 1986-06-06 1988-01-07 Transgene S.A. Verfahren zur Herstellung von Faktor VIII aus Säugerzellen
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
IL83192A (en) * 1986-07-18 1992-11-15 Gist Brocades Nv Method for the preparation of proteins with factor viii activity by microbial host cells;expression vectors,host cells,antibodies
IL84168A0 (en) * 1986-10-15 1988-03-31 Rorer Int Overseas Human factor viii-c analogs,process for the preparation thereof and pharmaceutical compositions containing the same
US4980456A (en) * 1987-04-06 1990-12-25 Scripps Clinic And Research Foundation Recombinant factor VIIIC derived fragments
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
JPH025890A (ja) 1988-06-24 1990-01-10 Chemo Sero Therapeut Res Inst ニワトリのβ−アクチンプロモーターを用いたヒトファクター8Cの製造方法
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning

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ES2169095T3 (es) 2002-07-01
DE3855523T2 (de) 1997-02-06
US6316226B1 (en) 2001-11-13
DE3856505D1 (de) 2002-01-10
FI890643A0 (fi) 1989-02-10
CN1033760C (zh) 1997-01-08
WO1988009813A1 (en) 1988-12-15
FI103731B1 (fi) 1999-08-31
FI103731B (fi) 1999-08-31
CA1341208C (en) 2001-03-27
NO890587D0 (no) 1989-02-10
PT87721A (pt) 1988-07-01
CN1030609A (zh) 1989-01-25
HU211503A9 (en) 1995-11-28
GR3021545T3 (en) 1997-02-28
ES2094111T3 (es) 1997-01-16
FI890643L (fi) 1989-02-10
IE881755L (en) 1988-12-12
NO301121B1 (no) 1997-09-15
IL86693A0 (en) 1988-11-30
ZA884216B (en) 1989-02-22
DE3855523D1 (de) 1996-10-17
PT87721B (pt) 1992-10-30
JPH02500484A (ja) 1990-02-22
DK172631B1 (da) 1999-03-22
HU213300B (en) 1997-05-28
NO890587L (no) 1989-02-10
IE69026B1 (en) 1996-08-07
DE3856505T2 (de) 2002-08-08
IL86693A (en) 1994-06-24
KR970002915B1 (ko) 1997-03-12
NZ224992A (en) 1991-07-26
US5171844A (en) 1992-12-15
DK58789A (da) 1989-02-09
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ATE142691T1 (de) 1996-09-15
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