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ATE417618T1 - Faktor-viii-polypeptide auf hohem expressionsniveau codierende nukleinsäure- und aminosäuresequenzen und verwendungsverfahren - Google Patents

Faktor-viii-polypeptide auf hohem expressionsniveau codierende nukleinsäure- und aminosäuresequenzen und verwendungsverfahren

Info

Publication number
ATE417618T1
ATE417618T1 AT02773804T AT02773804T ATE417618T1 AT E417618 T1 ATE417618 T1 AT E417618T1 AT 02773804 T AT02773804 T AT 02773804T AT 02773804 T AT02773804 T AT 02773804T AT E417618 T1 ATE417618 T1 AT E417618T1
Authority
AT
Austria
Prior art keywords
methods
factor viii
amino acid
nucleic acid
expression level
Prior art date
Application number
AT02773804T
Other languages
English (en)
Inventor
John Lollar
Original Assignee
Expression Therapeutics 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 Expression Therapeutics Llc filed Critical Expression Therapeutics Llc
Application granted granted Critical
Publication of ATE417618T1 publication Critical patent/ATE417618T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AT02773804T 2001-10-05 2002-10-07 Faktor-viii-polypeptide auf hohem expressionsniveau codierende nukleinsäure- und aminosäuresequenzen und verwendungsverfahren ATE417618T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US32738801P 2001-10-05 2001-10-05

Publications (1)

Publication Number Publication Date
ATE417618T1 true ATE417618T1 (de) 2009-01-15

Family

ID=23276347

Family Applications (1)

Application Number Title Priority Date Filing Date
AT02773804T ATE417618T1 (de) 2001-10-05 2002-10-07 Faktor-viii-polypeptide auf hohem expressionsniveau codierende nukleinsäure- und aminosäuresequenzen und verwendungsverfahren

Country Status (9)

Country Link
US (3) US7635763B2 (de)
EP (1) EP1572889B1 (de)
JP (2) JP4634036B2 (de)
AT (1) ATE417618T1 (de)
AU (1) AU2002337901B2 (de)
CA (1) CA2461443C (de)
DE (1) DE60230456D1 (de)
ES (1) ES2319745T3 (de)
WO (1) WO2003031598A2 (de)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7560107B2 (en) 1996-06-26 2009-07-14 Emory University Modified factor VIII
DE60230456D1 (de) * 2001-10-05 2009-01-29 Expression Therapeutics Llc Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahren
US7485291B2 (en) 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
EP1636360A4 (de) 2003-06-03 2006-11-08 Cell Genesys Inc Zusammensetzungen und verfahren zur verstärkten expression rekombinanter polypeptide von einem vektor unter verwendung einer peptidspaltstelle
PT1750733E (pt) * 2004-05-03 2014-02-14 Baxter Healthcare Sa Método de administração de factor viii suíno sem domínio b
PT2363414T (pt) 2004-11-12 2022-08-04 Bayer Healthcare Llc Modificação de fviii direcionada a sítio
WO2009140598A1 (en) * 2008-05-16 2009-11-19 Bayer Healthcare Llc Targeted coagulation factors and method of using the same
MX351220B (es) * 2010-11-05 2017-10-05 Baxalta GmbH Una variante nueva del factor viii antihemofilico que tiene actividad especifica incrementada.
ES2913994T3 (es) 2011-07-08 2022-06-07 Bioverativ Therapeutics Inc Polipéptidos quiméricos e híbridos del Factor VIII, y métodos de uso de los mismos
WO2014008172A2 (en) * 2012-07-03 2014-01-09 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
US20160102133A1 (en) 2013-06-24 2016-04-14 Weidong Xiao Mutant factor viii compositions and methods
US10058624B2 (en) 2015-04-16 2018-08-28 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
SG10202106307UA (en) 2015-11-13 2021-07-29 Takeda Pharmaceuticals Co Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
CN108602876B (zh) 2015-11-13 2022-07-05 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
EP3452102B9 (de) 2016-04-15 2024-09-04 The Trustees of The University of Pennsylvania Gentherapie zur behandlung von hämophilie a
WO2019040743A1 (en) 2017-08-23 2019-02-28 Wake Forest University Health Sciences POST-NATAL TRANSPLANTATION OF CELLS EXPRESSING FACTOR VIII FOR THE TREATMENT OF HEMOPHILIA
WO2019040747A1 (en) 2017-08-23 2019-02-28 Wake Forest University Health Sciences IN UTERO TRANSPLANTATION OF CELLS EXPRESSING FACTOR VIII FOR THE TREATMENT OF HEMOPHILIA
KR20210034013A (ko) 2018-07-16 2021-03-29 박스알타 인코퍼레이티드 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법
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
US11680109B2 (en) 2019-05-29 2023-06-20 Duke University Compositions and methods for detection and measurement of RNA modifications through targeted RNA editing
EP4284412A4 (de) * 2021-01-29 2025-04-09 Expression Therapeutics, LLC Nukleinsäure- und aminosäuresequenzen zur codierung von high-level-expressor-faktor-viii-polypeptiden und verfahren zur verwendung

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6376463B1 (en) * 1992-04-07 2002-04-23 Emory University Modified factor VIII
US5859204A (en) * 1992-04-07 1999-01-12 Emory University Modified factor VIII
US5364771A (en) * 1992-04-07 1994-11-15 Emory University Hybrid human/porcine factor VIII
US5663060A (en) * 1992-04-07 1997-09-02 Emory University Hybrid human/animal factor VIII
US5888974A (en) * 1992-04-07 1999-03-30 Emory University Hybrid human/animal factor VIII
US5744446A (en) * 1992-04-07 1998-04-28 Emory University Hybrid human/animal factor VIII
US6180371B1 (en) * 1996-06-26 2001-01-30 Emory University Modified factor VIII
JPH08511423A (ja) * 1993-06-10 1996-12-03 ジェネティック セラピー,インコーポレイテッド 血友病治療のためのアデノウイルスベクター
US6818439B1 (en) * 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
US6458563B1 (en) * 1996-06-26 2002-10-01 Emory University Modified factor VIII
WO1999029848A1 (en) * 1997-12-05 1999-06-17 The Immune Response Corporation Novel vectors and genes exhibiting increased expression
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
DE60230456D1 (de) * 2001-10-05 2009-01-29 Expression Therapeutics Llc Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahren

Also Published As

Publication number Publication date
CA2461443C (en) 2011-07-12
US8188246B2 (en) 2012-05-29
US20120270266A1 (en) 2012-10-25
US20050118684A1 (en) 2005-06-02
JP5162635B2 (ja) 2013-03-13
WO2003031598A3 (en) 2006-01-05
JP4634036B2 (ja) 2011-02-16
AU2002337901B2 (en) 2008-01-17
US7635763B2 (en) 2009-12-22
CA2461443A1 (en) 2003-04-17
JP2011015698A (ja) 2011-01-27
EP1572889A4 (de) 2006-09-06
WO2003031598A2 (en) 2003-04-17
HK1080517A1 (en) 2006-04-28
US8519111B2 (en) 2013-08-27
DE60230456D1 (de) 2009-01-29
US20110077203A1 (en) 2011-03-31
JP2005518783A (ja) 2005-06-30
EP1572889B1 (de) 2008-12-17
EP1572889A2 (de) 2005-09-14
ES2319745T3 (es) 2009-05-12

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