KR102404550B1 - A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 - Google Patents

A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 Download PDF

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KR102404550B1
KR102404550B1 KR1020187015191A KR20187015191A KR102404550B1 KR 102404550 B1 KR102404550 B1 KR 102404550B1 KR 1020187015191 A KR1020187015191 A KR 1020187015191A KR 20187015191 A KR20187015191 A KR 20187015191A KR 102404550 B1 KR102404550 B1 KR 102404550B1
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ser
seq
factor viii
glu
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팔코-귄터 팔크너
프란치스카 호링
조하네스 렝글러
한스페터 로텐스타이너
프리드리히 샤이프링거
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다케다 야쿠힌 고교 가부시키가이샤
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020187015191A 2015-11-13 2016-11-11 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 Active KR102404550B1 (ko)

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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

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KR20180070700A KR20180070700A (ko) 2018-06-26
KR102404550B1 true KR102404550B1 (ko) 2022-05-31

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

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130017997A1 (en) 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
US20150071883A1 (en) 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

Family Cites Families (39)

* 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
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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130017997A1 (en) 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
US20150071883A1 (en) 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

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KR20180070700A (ko) 2018-06-26
MX2018005969A (es) 2018-11-29
EA201891138A1 (ru) 2018-12-28
EA202092049A1 (ru) 2020-11-10
TWI850587B (zh) 2024-08-01
MY205187A (en) 2024-10-04
ZA201803442B (en) 2019-07-31
CN108602876A (zh) 2018-09-28
BR112018009732A8 (pt) 2019-02-26
AU2016354550A1 (en) 2018-06-07
CO2018005380A2 (es) 2018-05-31
IL259295A (en) 2018-07-31
EP3374388A1 (en) 2018-09-19
CL2018001301A1 (es) 2018-12-28
EA036944B1 (ru) 2021-01-19
AU2016354550B2 (en) 2020-04-16
CL2020001611A1 (es) 2020-10-30
TW202229555A (zh) 2022-08-01
US20170226188A1 (en) 2017-08-10
JP6695426B2 (ja) 2020-05-20
US10189889B2 (en) 2019-01-29
NZ742555A (en) 2023-12-22
IL259295B1 (en) 2024-06-01
CN108602876B (zh) 2022-07-05
TW201723180A (zh) 2017-07-01
BR112018009732A2 (en) 2018-11-21
IL259295B2 (en) 2024-10-01
JP2018537089A (ja) 2018-12-20
CA3005565A1 (en) 2017-05-18
SG11201804064WA (en) 2018-06-28
TWI823830B (zh) 2023-12-01
WO2017083764A1 (en) 2017-05-18

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