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CL2018001299A1 - Vectores virales que codifican variantes de la fviii recombinante con expresión aumentada para la terapia genetica de la hemofilia a. - Google Patents

Vectores virales que codifican variantes de la fviii recombinante con expresión aumentada para la terapia genetica de la hemofilia a.

Info

Publication number
CL2018001299A1
CL2018001299A1 CL2018001299A CL2018001299A CL2018001299A1 CL 2018001299 A1 CL2018001299 A1 CL 2018001299A1 CL 2018001299 A CL2018001299 A CL 2018001299A CL 2018001299 A CL2018001299 A CL 2018001299A CL 2018001299 A1 CL2018001299 A1 CL 2018001299A1
Authority
CL
Chile
Prior art keywords
hemophilia
viral vectors
increased expression
vectors encoding
recombinant fviii
Prior art date
Application number
CL2018001299A
Other languages
English (en)
Inventor
Falko-Günter Falkner
Franziska Horling
Johannes Lengler
Hanspeter Rottensteiner
Friedrich Scheiflinger
Original Assignee
Baxalta Inc
Baxalta GmbH
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of CL2018001299A1 publication Critical patent/CL2018001299A1/es

Links

Classifications

    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CL2018001299A 2015-11-13 2018-05-14 Vectores virales que codifican variantes de la fviii recombinante con expresión aumentada para la terapia genetica de la hemofilia a. CL2018001299A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562255317P 2015-11-13 2015-11-13

Publications (1)

Publication Number Publication Date
CL2018001299A1 true CL2018001299A1 (es) 2019-03-15

Family

ID=57539605

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2018001299A CL2018001299A1 (es) 2015-11-13 2018-05-14 Vectores virales que codifican variantes de la fviii recombinante con expresión aumentada para la terapia genetica de la hemofilia a.

Country Status (18)

Country Link
US (4) US10189888B2 (es)
EP (1) EP3374387A1 (es)
JP (4) JP6768798B2 (es)
KR (2) KR102484553B1 (es)
CN (3) CN108884145B (es)
AU (4) AU2016353353B2 (es)
BR (1) BR112018009717B1 (es)
CA (1) CA3005334A1 (es)
CL (1) CL2018001299A1 (es)
CO (1) CO2018005377A2 (es)
EA (2) EA038288B1 (es)
IL (3) IL281038B2 (es)
MX (1) MX2018005982A (es)
MY (1) MY190067A (es)
SG (2) SG10202106307UA (es)
TW (3) TW202302627A (es)
WO (1) WO2017083762A1 (es)
ZA (3) ZA201803443B (es)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3368664A4 (en) 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
TWI850587B (zh) 2015-11-13 2024-08-01 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變異體之病毒載體
MY190067A (en) 2015-11-13 2022-03-24 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EP3442597A1 (en) 2016-04-15 2019-02-20 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia b
CN109562191A (zh) * 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 用于治疗血友病a的基因疗法
ES2981519T3 (es) 2016-11-04 2024-10-09 Takeda Pharmaceuticals Co Formulaciones de virus adeno-asociados
US20200263196A1 (en) * 2017-09-27 2020-08-20 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
KR102105145B1 (ko) * 2017-10-13 2020-05-13 영남대학교 산학협력단 촬영 장치 및 용기
KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
BR112021000695A2 (pt) * 2018-07-16 2021-04-20 Baxalta Incorporated métodos para tratar hemofilia a e para monitorar a eficácia da terapia gênica de fator viii de hemofilia a.
EP3829650A4 (en) * 2018-08-03 2022-05-04 Sangamo Therapeutics, Inc. CLINICAL PARAMETERS IMPROVED BY FACTOR VIII EXPRESSION
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
CA3129532A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
WO2020257586A2 (en) * 2019-06-20 2020-12-24 Baxalta Incorporated Method of treatment with viral-based gene therapy
CN114829389A (zh) * 2019-11-01 2022-07-29 自由行疗法有限公司 转录调节元件
CN114981299A (zh) * 2019-12-12 2022-08-30 武田药品工业株式会社 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法
KR20220138346A (ko) * 2021-04-02 2022-10-12 연세대학교 산학협력단 Fe-fviii 변이 유전자로 교정된 줄기세포, 이로부터 분화된 내피 세포 및 이를 포함하는 혈우병 예방 또는 치료용 약제학적 조성물
WO2023211316A1 (en) * 2022-04-28 2023-11-02 Joint Stock Company "Biocad" Codon-optimized nucleic acid encoding the fviii-bdd
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2024191428A1 (en) 2023-03-14 2024-09-19 Sigilon Therapeutics, Inc. Hydrogel capsules comprising covalently photocrosslinked polysaccharides and islet cells

Family Cites Families (53)

* 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
CA1341174C (en) 1985-04-12 2001-01-30 John J. Toole Jr. Procoagulant proteins derived from factor viii: c
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
US5610278A (en) 1986-06-24 1997-03-11 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
US5171844A (en) 1987-06-12 1992-12-15 Gist-Brocades N.W. Proteins with factor viii activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
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
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
ATE322547T1 (de) 1993-06-10 2006-04-15 Genetic Therapy Inc Adenovirale vektoren für die behandlung der hämophilie
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
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
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
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
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human 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.
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
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
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
EP2037892B1 (en) 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
KR101542752B1 (ko) 2006-12-22 2015-08-10 체에스엘 베링 게엠베하 연장된 생체내 반감기를 갖는 변형된 응고 인자
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
US20120093840A1 (en) 2009-04-06 2012-04-19 Novo Nordisk A/S Targeted delivery of factor viii proteins to platelets
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
MX2013000301A (es) 2010-07-09 2013-05-09 Biogen Idec Hemophilia Inc Factores quimericos de coagulacion.
US20130017997A1 (en) 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
EA028914B1 (ru) 2011-07-25 2018-01-31 Байоджен Хемофилия Инк. Исследования для мониторинга нарушений свертываемости крови
EA035323B1 (ru) 2012-01-12 2020-05-28 Биовератив Терапьютикс Инк. Полипептиды химерного фактора viii и их применение
CN104254614A (zh) * 2012-01-12 2014-12-31 比奥根艾迪克Ma公司 降低经受因子viii疗法的个体中针对因子viii的免疫原性的方法
KR102097263B1 (ko) * 2012-02-15 2020-04-06 바이오버라티브 테라퓨틱스 인크. Viii 인자 조성물 및 이를 제조하고 사용하는 방법
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 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
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
EP3889173B1 (en) 2013-02-15 2023-07-05 Bioverativ Therapeutics Inc. Optimized factor viii gene
KR20150132449A (ko) 2013-03-15 2015-11-25 바이엘 헬스케어 엘엘씨 재조합 인자 viii 제제
DK3013855T3 (da) * 2013-06-24 2021-01-11 Xiao Weidong Mutante faktor VIII-sammensætninger og fremgangsmåder
WO2015038625A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
BR112017002781A2 (pt) 2014-08-13 2017-12-19 Sabatino Denise cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia
JP6800863B2 (ja) * 2015-02-06 2020-12-16 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 最適化されたヒト凝固第ｖｉｉｉ因子遺伝子発現カセットおよびその使用
AU2016232146B2 (en) 2015-03-17 2021-11-04 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
MY190067A (en) 2015-11-13 2022-03-24 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

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Publication number Publication date
EA201891137A1 (ru) 2018-12-28
ZA201803443B (en) 2019-03-27
JP7069258B2 (ja) 2022-05-17
BR112018009717A2 (pt) 2018-11-21
JP7307836B2 (ja) 2023-07-12
JP6768798B2 (ja) 2020-10-14
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