ES2319745T3 - Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso. - Google Patents
Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso. Download PDFInfo
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- ES2319745T3 ES2319745T3 ES02773804T ES02773804T ES2319745T3 ES 2319745 T3 ES2319745 T3 ES 2319745T3 ES 02773804 T ES02773804 T ES 02773804T ES 02773804 T ES02773804 T ES 02773804T ES 2319745 T3 ES2319745 T3 ES 2319745T3
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Classifications
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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)
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- 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)
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- Veterinary Medicine (AREA)
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- 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)
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| US32738801P | 2001-10-05 | 2001-10-05 | |
| US327388P | 2001-10-05 |
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| ES2319745T3 true ES2319745T3 (es) | 2009-05-12 |
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| EP (1) | EP1572889B1 (enExample) |
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| DE (1) | DE60230456D1 (enExample) |
| ES (1) | ES2319745T3 (enExample) |
| WO (1) | WO2003031598A2 (enExample) |
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| 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 |
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| 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 |
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2002
- 2002-10-07 CA CA2461443A patent/CA2461443C/en not_active Expired - Lifetime
- 2002-10-07 JP JP2003534569A patent/JP4634036B2/ja not_active Expired - Lifetime
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- 2002-10-07 WO PCT/US2002/033403 patent/WO2003031598A2/en not_active Ceased
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2009
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| JP4634036B2 (ja) | 2011-02-16 |
| 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 |
| 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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