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

    • 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)
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal 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
ES2319745T3 (es) * 2001-10-05 2009-05-12 Expression Therapeutics, Llc Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso.
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
WO2005017149A1 (en) 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
DK1750733T3 (da) * 2004-05-03 2014-01-20 Univ Emory FREMGANGSMÅDE TIL INDGIVELSE AF SVINE-B-DOMÆNELØS fVIII
PT2363414T (pt) 2004-11-12 2022-08-04 Bayer Healthcare Llc Modificação de fviii direcionada a sítio
CN102112144A (zh) * 2008-05-16 2011-06-29 拜耳医药保健有限公司 靶向性凝固因子及其使用方法
DK2635297T3 (da) 2010-11-05 2019-05-06 Baxalta GmbH En ny variant af antihæmofil faktor viii, der har en øget specifik aktivitet
PT3513804T (pt) 2011-07-08 2022-06-02 Bioverativ Therapeutics Inc Polipéptidos fator viii híbridos e quiméricos, e métodos de utilização dos mesmos
US8986991B2 (en) * 2012-07-03 2015-03-24 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
LT3013855T (lt) * 2013-06-24 2021-01-25 Xiao, Weidong Mutavusio viii faktoriaus kompozicijos ir metodai
EP3283126B1 (de) * 2015-04-16 2019-11-06 Emory University Rekombinante promotoren und vektoren zur proteinexpression in leber und verwendung davon
CA3005565A1 (en) 2015-11-13 2017-05-18 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EA202190827A1 (ru) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
MX2018012537A (es) 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
US20200368286A1 (en) 2017-08-23 2020-11-26 Wake Forest University Health Sciences In utero transplantation of factor viii-expressing cells for treatment of hemophilia
CA3073678A1 (en) 2017-08-23 2019-02-28 Children's Healthcare Of Atlanta, Inc. Post-natal transplantation of factor viii-expressing cells for treatment of hemophilia
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
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
US20230357360A1 (en) * 2021-01-29 2023-11-09 Expression Therapeutics, Llc Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5744446A (en) * 1992-04-07 1998-04-28 Emory University Hybrid human/animal 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
US6180371B1 (en) * 1996-06-26 2001-01-30 Emory University Modified factor VIII
US6376463B1 (en) * 1992-04-07 2002-04-23 Emory University Modified factor VIII
EP0710288B1 (de) * 1993-06-10 2006-04-05 Genetic Therapy, Inc. Adenovirale vektoren für die behandlung der hämophilie
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
EP1036177A1 (de) * 1997-12-05 2000-09-20 The Immune Response Corporation Vektoren und gene die eine verbesserte genexpression zur folge haben
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
ES2319745T3 (es) * 2001-10-05 2009-05-12 Expression Therapeutics, Llc Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso.

Also Published As

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

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