JP4634036B2 - 高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法 - Google Patents

高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法 Download PDF

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JP4634036B2
JP4634036B2 JP2003534569A JP2003534569A JP4634036B2 JP 4634036 B2 JP4634036 B2 JP 4634036B2 JP 2003534569 A JP2003534569 A JP 2003534569A JP 2003534569 A JP2003534569 A JP 2003534569A JP 4634036 B2 JP4634036 B2 JP 4634036B2
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factor viii
polypeptide
nucleic acid
seq
sequence
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JP2005518783A5 (enExample
JP2005518783A (ja
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ジョン エス． ロラー，
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エクスプレッション セラピューティクス， エルエルシー
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    • 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

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  • 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)
JP2003534569A 2001-10-05 2002-10-07 高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法 Expired - Lifetime JP4634036B2 (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US32738801P 2001-10-05 2001-10-05
PCT/US2002/033403 WO2003031598A2 (en) 2001-10-05 2002-10-07 Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use

Related Child Applications (1)

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JP2010210204A Division JP5162635B2 (ja) 2001-10-05 2010-09-17 高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法

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JP2005518783A JP2005518783A (ja) 2005-06-30
JP2005518783A5 JP2005518783A5 (enExample) 2006-01-12
JP4634036B2 true JP4634036B2 (ja) 2011-02-16

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JP2003534569A Expired - Lifetime JP4634036B2 (ja) 2001-10-05 2002-10-07 高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法
JP2010210204A Expired - Lifetime JP5162635B2 (ja) 2001-10-05 2010-09-17 高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法

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JP2010210204A Expired - Lifetime JP5162635B2 (ja) 2001-10-05 2010-09-17 高レベルエキスプレッサー第ｖｉｉｉ因子ポリペプチドをコードする核酸配列およびアミノ酸配列および使用方法

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

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
CA2461443C (en) * 2001-10-05 2011-07-12 Emory University Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
CA2526120A1 (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
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
WO2005107776A1 (en) 2004-05-03 2005-11-17 Emory University METHOD OF ADMINISTERING PORCINE B-DOMAINLESS fVIII
KR101483917B1 (ko) 2004-11-12 2015-01-16 바이엘 헬스케어 엘엘씨 Ｆｖｉｉｉ의 부위 지향 변형
JP5997443B2 (ja) * 2008-05-16 2016-09-28 バイエル・ヘルスケア・エルエルシーＢａｙｅｒ ＨｅａｌｔｈＣａｒｅ ＬＬＣ 標的化凝固因子およびそれを使用する方法
CA2816575C (en) * 2010-11-05 2019-06-11 Baxter Healthcare S.A. A new variant of antihemophilic factor viii having increased specific activity
EP2729161B1 (en) * 2011-07-08 2018-12-19 Bioverativ Therapeutics Inc. Factor viii chimeric and hybrid polypeptides, and methods of use thereof
WO2014008172A2 (en) * 2012-07-03 2014-01-09 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
RS61305B1 (sr) * 2013-06-24 2021-02-26 Xiao Weidong Preparati i metode mutantskog faktora viii
ES3009565T3 (en) 2015-04-16 2025-03-27 Univ Emory Recombinant promoters and vectors for protein expression in liver and use thereof
AU2016353353B2 (en) 2015-11-13 2020-07-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
EA036944B1 (ru) 2015-11-13 2021-01-19 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
SG11201808422QA (en) 2016-04-15 2018-10-30 Univ Pennsylvania Gene therapy for treating hemophilia a
CA3073678A1 (en) 2017-08-23 2019-02-28 Wake Forest University Health Sciences Post-natal transplantation of factor viii-expressing cells for 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
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
US5663060A (en) 1992-04-07 1997-09-02 Emory University Hybrid human/animal factor VIII
US6180371B1 (en) 1996-06-26 2001-01-30 Emory University Modified factor VIII
US5859204A (en) 1992-04-07 1999-01-12 Emory University Modified factor VIII
US5744446A (en) 1992-04-07 1998-04-28 Emory University Hybrid human/animal factor VIII
US5888974A (en) 1992-04-07 1999-03-30 Emory University Hybrid human/animal factor VIII
US5364771A (en) 1992-04-07 1994-11-15 Emory University Hybrid human/porcine factor VIII
US6376463B1 (en) 1992-04-07 2002-04-23 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
CA2461443C (en) * 2001-10-05 2011-07-12 Emory University Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use

Also Published As

Publication number Publication date
US20110077203A1 (en) 2011-03-31
HK1080517A1 (en) 2006-04-28
ATE417618T1 (de) 2009-01-15
CA2461443A1 (en) 2003-04-17
US8519111B2 (en) 2013-08-27
EP1572889A2 (en) 2005-09-14
ES2319745T3 (es) 2009-05-12
JP2011015698A (ja) 2011-01-27
JP2005518783A (ja) 2005-06-30
JP5162635B2 (ja) 2013-03-13
CA2461443C (en) 2011-07-12
US20050118684A1 (en) 2005-06-02
US20120270266A1 (en) 2012-10-25
EP1572889A4 (en) 2006-09-06
US8188246B2 (en) 2012-05-29
DE60230456D1 (de) 2009-01-29
AU2002337901B2 (en) 2008-01-17
US7635763B2 (en) 2009-12-22
WO2003031598A2 (en) 2003-04-17
WO2003031598A3 (en) 2006-01-05
EP1572889B1 (en) 2008-12-17

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