JP2018537089A5 - - Google Patents

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Publication number
JP2018537089A5
JP2018537089A5 JP2018524806A JP2018524806A JP2018537089A5 JP 2018537089 A5 JP2018537089 A5 JP 2018537089A5 JP 2018524806 A JP2018524806 A JP 2018524806A JP 2018524806 A JP2018524806 A JP 2018524806A JP 2018537089 A5 JP2018537089 A5 JP 2018537089A5
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JP
Japan
Prior art keywords
polynucleotide
nucleotide sequence
seq
factor viii
aav
Prior art date
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JP2018524806A
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English (en)
Japanese (ja)
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JP6695426B2 (ja
JP2018537089A (ja
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Priority claimed from PCT/US2016/061688 external-priority patent/WO2017083764A1/en
Publication of JP2018537089A publication Critical patent/JP2018537089A/ja
Publication of JP2018537089A5 publication Critical patent/JP2018537089A5/ja
Application granted granted Critical
Publication of JP6695426B2 publication Critical patent/JP6695426B2/ja
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JP2018524806A 2015-11-13 2016-11-11 組換えfviii変異型をコードする血友病a遺伝子治療用高発現ウイルスベクター Active JP6695426B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255323P 2015-11-13 2015-11-13
US62/255,323 2015-11-13
PCT/US2016/061688 WO2017083764A1 (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Publications (3)

Publication Number Publication Date
JP2018537089A JP2018537089A (ja) 2018-12-20
JP2018537089A5 true JP2018537089A5 (enExample) 2019-12-19
JP6695426B2 JP6695426B2 (ja) 2020-05-20

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ID=57539606

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018524806A Active JP6695426B2 (ja) 2015-11-13 2016-11-11 組換えfviii変異型をコードする血友病a遺伝子治療用高発現ウイルスベクター

Country Status (18)

Country Link
US (1) US10189889B2 (enExample)
EP (1) EP3374388A1 (enExample)
JP (1) JP6695426B2 (enExample)
KR (1) KR102404550B1 (enExample)
CN (1) CN108602876B (enExample)
AU (1) AU2016354550B2 (enExample)
BR (1) BR112018009732A8 (enExample)
CA (1) CA3005565A1 (enExample)
CL (2) CL2018001301A1 (enExample)
CO (1) CO2018005380A2 (enExample)
EA (2) EA036944B1 (enExample)
IL (1) IL259295B2 (enExample)
MX (1) MX2018005969A (enExample)
MY (1) MY205187A (enExample)
SG (1) SG11201804064WA (enExample)
TW (2) TWI823830B (enExample)
WO (1) WO2017083764A1 (enExample)
ZA (1) ZA201803442B (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
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
SG11202108357PA (en) 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
CN109735547B (zh) * 2019-03-19 2022-05-31 青岛蔚蓝生物集团有限公司 一种提高毕赤酵母外源蛋白表达量的启动子及其应用
CN114375205A (zh) * 2019-06-20 2022-04-19 武田药品工业株式会社 用基于病毒的基因疗法进行治疗的方法
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

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
ES8801674A1 (es) 1985-04-12 1988-02-16 Genetics Inst Un procedimiento para la preparacion de una proteina que presenta actividad procoagulante.
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
US6060447A (en) 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US6346513B1 (en) 1987-06-12 2002-02-12 Baxter Trading Gmbh Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
IL86693A (en) 1987-06-12 1994-06-24 Stichting Centraal Lab Proteins with factor VIII activity, process for their preparation using genetically engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
EP0710288B1 (en) 1993-06-10 2006-04-05 Genetic Therapy, Inc. Adenoviral vectors for treatment of hemophilia
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
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
CN1179976C (zh) * 2000-12-29 2004-12-15 中国科学院上海生物化学研究所 产生凝血因子ⅷ的生产方法和宿主细胞
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.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
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
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US20130017997A1 (en) * 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
EA038705B1 (ru) * 2012-01-12 2021-10-07 Биовератив Терапьютикс Инк. Способы снижения иммуногенности фактора свёртывания крови viii у пациентов, получающих лечение фактором viii
JP6256882B2 (ja) * 2012-02-15 2018-01-10 アムニクス オペレーティング インコーポレイテッド 第viii因子組成物、ならびに組成物の作製方法および用途
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
US8986991B2 (en) 2012-07-03 2015-03-24 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
US10398787B2 (en) 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
CN107427557B (zh) 2014-08-13 2022-01-04 费城儿童医院 用于包装和表达变体因子viii以治疗血友病的改良表达组件
US11007280B2 (en) 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
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

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