JP2015521465A5 - - Google Patents
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- Publication number
- JP2015521465A5 JP2015521465A5 JP2015516684A JP2015516684A JP2015521465A5 JP 2015521465 A5 JP2015521465 A5 JP 2015521465A5 JP 2015516684 A JP2015516684 A JP 2015516684A JP 2015516684 A JP2015516684 A JP 2015516684A JP 2015521465 A5 JP2015521465 A5 JP 2015521465A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- nucleic acid
- acid molecule
- seq
- factor viii
- Prior art date
- Legal status (The legal status 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 status listed.)
- Granted
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015521465A JP2015521465A (ja) | 2015-07-30 |
| JP2015521465A5 true JP2015521465A5 (https=) | 2016-08-04 |
| JP6473416B2 JP6473416B2 (ja) | 2019-02-20 |
Family
ID=46605791
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015516684A Expired - Fee Related JP6473416B2 (ja) | 2012-06-12 | 2013-06-12 | 第ｖｉｉｉ因子配列 |
Country Status (7)
| Country | Link |
|---|---|
| US (6) | US9447168B2 (https=) |
| EP (1) | EP2859016B1 (https=) |
| JP (1) | JP6473416B2 (https=) |
| AU (1) | AU2013276247B2 (https=) |
| ES (1) | ES2640586T3 (https=) |
| GB (1) | GB201210357D0 (https=) |
| WO (1) | WO2013186563A2 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| DK3080274T3 (da) | 2013-12-09 | 2020-08-31 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til genom-manipulation |
| CN115074366A (zh) * | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| RU2742352C2 (ru) | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3374387A1 (en) | 2015-11-13 | 2018-09-19 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| EA202092049A1 (ru) * | 2015-11-13 | 2020-11-10 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| PE20190401A1 (es) | 2016-07-26 | 2019-03-13 | Biomarin Pharm Inc | Novedosas proteinas de la capside del virus adenoasociado |
| BR112020022722A8 (pt) | 2018-05-09 | 2022-01-18 | Biomarin Pharm Inc | Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método |
| 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 |
| US20200069819A1 (en) | 2018-05-14 | 2020-03-05 | Biomarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
| EP3823985A1 (en) | 2018-07-16 | 2021-05-26 | Baxalta Incorporated | 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 |
| US11529427B2 (en) * | 2019-02-15 | 2022-12-20 | Crispr Therapeutics Ag | Gene editing for hemophilia A with improved factor VIII expression |
| 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 |
| US20230407328A1 (en) | 2020-11-02 | 2023-12-21 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| EP4355768A1 (en) | 2021-06-14 | 2024-04-24 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| JP2025504404A (ja) | 2022-01-14 | 2025-02-12 | アンジャリウム バイオサイエンシズ エージー | Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法 |
| WO2024007978A1 (zh) | 2022-07-07 | 2024-01-11 | 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 |
| TW202517669A (zh) | 2023-07-05 | 2025-05-01 | 日商武田藥品工業股份有限公司 | 用於Ａ型血友病之基因療法的編碼表現增加之重組ＦＶｌｌｌ變異體的病毒載體 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US6383794B1 (en) | 1998-08-24 | 2002-05-07 | Uab Research Foundation | Methods of producing high titer recombinant adeno-associated virus |
| 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 |
| US6103526A (en) | 1998-10-08 | 2000-08-15 | Protein Sciences Corporation | Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| KR100581574B1 (ko) | 2000-03-22 | 2006-05-22 | 옥타게네 게엠베하 | 인간 세포주 내 재조합 혈액 응고 인자의 제조 |
| US7351813B2 (en) | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| GB0325379D0 (en) | 2003-10-30 | 2003-12-03 | Oxford Biomedica Ltd | Vectors |
| EP1804839B1 (en) | 2004-09-22 | 2012-03-14 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| CN1286981C (zh) | 2004-11-30 | 2006-11-29 | 华中科技大学同济医学院附属同济医院 | 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法 |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US20090175833A1 (en) | 2006-01-13 | 2009-07-09 | Paula Dore-Duffy | Pericytes for use as stem cells |
| EP2848253A1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor VIII and factor IX genes and vectors for gene therapy |
| KR101589259B1 (ko) | 2006-06-21 | 2016-02-01 | 유니큐어 아이피 비.브이. | 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터 |
| SI3093345T1 (sl) | 2007-07-26 | 2019-08-30 | Uniqure Ip B.V. | Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami |
| WO2009017533A1 (en) | 2007-07-27 | 2009-02-05 | Porteon Electric Vehicles, Inc. | Modular light vehicle chassis |
| WO2010111414A1 (en) * | 2009-03-24 | 2010-09-30 | Bayer Healthcare Llc | Factor viii variants and methods of use |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| WO2011139629A2 (en) | 2010-04-26 | 2011-11-10 | Biogen Idec Ma Inc. | Light targeting molecules and uses thereof |
| GB201210357D0 (en) * | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| RS61039B1 (sr) | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
-
2012
- 2012-06-12 GB GBGB1210357.8A patent/GB201210357D0/en not_active Ceased
-
2013
- 2013-06-12 JP JP2015516684A patent/JP6473416B2/ja not_active Expired - Fee Related
- 2013-06-12 ES ES13728823.9T patent/ES2640586T3/es active Active
- 2013-06-12 AU AU2013276247A patent/AU2013276247B2/en active Active
- 2013-06-12 US US14/407,008 patent/US9447168B2/en active Active
- 2013-06-12 EP EP13728823.9A patent/EP2859016B1/en active Active
- 2013-06-12 WO PCT/GB2013/051551 patent/WO2013186563A2/en not_active Ceased
-
2016
- 2016-09-01 US US15/254,984 patent/US10124041B2/en active Active
-
2018
- 2018-07-10 US US16/031,503 patent/US10792336B2/en active Active
-
2020
- 2020-09-02 US US17/010,595 patent/US11419920B2/en active Active
-
2022
- 2022-08-03 US US17/817,261 patent/US20230241180A1/en not_active Abandoned
-
2024
- 2024-09-09 US US18/828,841 patent/US20250108093A1/en active Pending
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