CA2875449A1 - Factor viii sequences - Google Patents
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- CA2875449A1 CA2875449A1 CA2875449A CA2875449A CA2875449A1 CA 2875449 A1 CA2875449 A1 CA 2875449A1 CA 2875449 A CA2875449 A CA 2875449A CA 2875449 A CA2875449 A CA 2875449A CA 2875449 A1 CA2875449 A1 CA 2875449A1
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- factor viii
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- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
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- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- 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
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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- A—HUMAN NECESSITIES
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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| GBGB1210357.8A GB201210357D0 (en) | 2012-06-12 | 2012-06-12 | Factor VIII sequences |
| GB1210357.8 | 2012-06-12 | ||
| PCT/GB2013/051551 WO2013186563A2 (en) | 2012-06-12 | 2013-06-12 | Factor viii sequences |
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| CA2875449A Pending CA2875449A1 (en) | 2012-06-12 | 2013-06-12 | Factor viii sequences |
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| ES (1) | ES2640586T3 (enExample) |
| GB (1) | GB201210357D0 (enExample) |
| WO (1) | WO2013186563A2 (enExample) |
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| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| CN105940013B (zh) | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| ES3009565T3 (en) * | 2015-04-16 | 2025-03-27 | Univ Emory | Recombinant promoters and vectors for protein expression in liver and use thereof |
| RU2020126034A (ru) | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| 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 |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| KR20210008491A (ko) | 2018-05-14 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 청소년 대상체에서 aav 벡터의 안정한 발현 |
| 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 |
| MA54951A (fr) | 2019-02-15 | 2021-12-22 | Bayer Healthcare Llc | Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée |
| BR112021015776A2 (pt) * | 2019-06-05 | 2021-11-09 | Bayer Healthcare Llc | Edição de gene para hemofilia a com expressão de fator viii melhorada |
| CN112575034B (zh) * | 2019-09-29 | 2023-04-25 | 济南赛尔生物科技股份有限公司 | 一种治疗a型血友病的产品及应用 |
| EP4073106A2 (en) | 2019-12-12 | 2022-10-19 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| MX2023005041A (es) | 2020-11-02 | 2023-05-17 | Biomarin Pharm Inc | Proceso para enriquecer virus adenoasociado. |
| US20240269241A1 (en) | 2021-06-14 | 2024-08-15 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| WO2023135273A2 (en) | 2022-01-14 | 2023-07-20 | Anjarium Biosciences Ag | Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof |
| CN119233998A (zh) | 2022-07-07 | 2024-12-31 | 深圳新诺微环生物科技有限公司 | 一种连接肽、包含连接肽的凝血八因子蛋白或其变体及其用途 |
| WO2024238591A2 (en) | 2023-05-15 | 2024-11-21 | Biomarin Pharmaceutical Inc. | Methods of treating anti-aav seropositive hemophilia patients |
| 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 |
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| US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
| US20090148906A1 (en) | 1998-09-29 | 2009-06-11 | Shire Human Genetic Therapies, Inc. A Delaware Corporation | Optimized messenger rna |
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| 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 |
| SI1266006T1 (sl) | 2000-03-22 | 2006-06-30 | Octagene Gmbh | Proizvodnja rekombinantnih muteinov krvnega koagulacijskega faktorja VIII v humanih celicnih linijah |
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| US11419920B2 (en) | 2022-08-23 |
| US20230241180A1 (en) | 2023-08-03 |
| US10124041B2 (en) | 2018-11-13 |
| EP2859016A2 (en) | 2015-04-15 |
| US10792336B2 (en) | 2020-10-06 |
| AU2013276247B2 (en) | 2017-03-09 |
| US20250108093A1 (en) | 2025-04-03 |
| JP6473416B2 (ja) | 2019-02-20 |
| ES2640586T3 (es) | 2017-11-03 |
| US9447168B2 (en) | 2016-09-20 |
| AU2013276247A1 (en) | 2015-01-15 |
| JP2015521465A (ja) | 2015-07-30 |
| US20170049859A1 (en) | 2017-02-23 |
| GB201210357D0 (en) | 2012-07-25 |
| US20180311317A1 (en) | 2018-11-01 |
| EP2859016B1 (en) | 2017-08-09 |
| US20200390866A1 (en) | 2020-12-17 |
| US20150158930A1 (en) | 2015-06-11 |
| WO2013186563A3 (en) | 2014-02-13 |
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