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SG10202106307UA - Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a - Google Patents

Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Info

Publication number
SG10202106307UA
SG10202106307UA SG10202106307UA SG10202106307UA SG10202106307UA SG 10202106307U A SG10202106307U A SG 10202106307UA SG 10202106307U A SG10202106307U A SG 10202106307UA SG 10202106307U A SG10202106307U A SG 10202106307UA SG 10202106307U A SG10202106307U A SG 10202106307UA
Authority
SG
Singapore
Prior art keywords
hemophilia
gene therapy
viral vectors
increased expression
vectors encoding
Prior art date
Application number
SG10202106307UA
Inventor
Falko-Günter Falkner
Franziska Horling
Johannes Lengler
Hanspeter Rottensteiner
Friedrich Scheiflinger
Original Assignee
Takeda Pharmaceuticals Co
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 Takeda Pharmaceuticals Co filed Critical Takeda Pharmaceuticals Co
Publication of SG10202106307UA publication Critical patent/SG10202106307UA/en

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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
SG10202106307UA 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a SG10202106307UA (en)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
SG10202106307UA true SG10202106307UA (en) 2021-07-29

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
SG10202106307UA SG10202106307UA (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201804070XA SG11201804070XA (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201804070XA SG11201804070XA (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Country Status (18)

Country Link
US (4) US10189888B2 (en)
EP (1) EP3374387A1 (en)
JP (4) JP6768798B2 (en)
KR (2) KR20230008256A (en)
CN (3) CN116479001A (en)
AU (4) AU2016353353B2 (en)
BR (1) BR112018009717B1 (en)
CA (1) CA3005334A1 (en)
CL (1) CL2018001299A1 (en)
CO (1) CO2018005377A2 (en)
EA (2) EA202190827A1 (en)
IL (3) IL305526A (en)
MX (1) MX2018005982A (en)
MY (2) MY198589A (en)
SG (2) SG10202106307UA (en)
TW (3) TW202302627A (en)
WO (1) WO2017083762A1 (en)
ZA (3) ZA201803443B (en)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
TWI850587B (en) * 2015-11-13 2024-08-01 日商武田藥品工業股份有限公司 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EA202190827A1 (en) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед VIRAL VECTORS CODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A
CA3019425A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia a
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
PT3534922T (en) 2016-11-04 2024-06-07 Takeda Pharmaceuticals Co Adeno-associated virus formulations
KR20200057051A (en) * 2017-09-27 2020-05-25 시질론 테라퓨틱스, 인크. Methods, compositions and implantable elements comprising active cells
US20200248179A1 (en) * 2017-10-02 2020-08-06 Research Institute At Nationwide Children's Hospital MiRNA Detargeting System for Tissue Specific Interference
KR102105145B1 (en) * 2017-10-13 2020-05-13 영남대학교 산학협력단 Photographing device and case
KR101952102B1 (en) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 Factor Ⅷ mutant expression vector with inhanced protein expression
CA3106590A1 (en) * 2018-07-16 2020-01-23 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
MX2021001375A (en) * 2018-08-03 2021-04-19 Sangamo Therapeutics Inc Improved clinical parameters by expression of factor viii.
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 (en) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
CA3129532A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
KR20220022110A (en) * 2019-06-05 2022-02-24 크리스퍼 테라퓨틱스 아게 Gene Editing for Hemophilia A Using Improved Factor VIII Expression
JP2022537555A (en) * 2019-06-20 2022-08-26 武田薬品工業株式会社 Virus-based gene therapy treatment methods
WO2021084277A2 (en) * 2019-11-01 2021-05-06 Freeline Therapeutics Limited Transcription regulatory elements
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
WO2022211604A1 (en) * 2021-04-02 2022-10-06 연세대학교 산학협력단 Stem cells edited with fe-fviii mutant gene, endothelial cells differentiated therefrom, and pharmaceutical composition containing same for prevention or treatment of hemophilia
KR102421131B1 (en) * 2021-05-28 2022-07-15 알엔에이진(주) Method for screening externally introduced mRNA that can exist for a long time in a cell
WO2023092864A1 (en) * 2021-11-25 2023-06-01 Sichuan Real&Best Biotech Co., Ltd. Engineered human fviii with enhanced secretion ability and clotting activity
MA68586A1 (en) * 2022-04-28 2025-04-30 Joint Stock Company "Biocad" CODON-OPTIMIZED NUCLEIC ACID ENCODING FVIII-BDD
CN115948408A (en) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 Improved human coagulation factor VIII gene expression cassette and application thereof
TW202435846A (en) 2023-03-14 2024-09-16 美商希吉隆醫療公司 Hydrogel capsules comprising covalently crosslinked polysaccharides and islet cells
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

Family Cites Families (54)

* 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
EP0218712B1 (en) 1985-04-12 1992-02-26 Genetics Institute, Inc. Novel procoagulant proteins
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 (en) 1987-08-11 1990-06-15 Transgene Sa FACTOR VIII ANALOG, PREPARATION METHOD AND PHARMACEUTICAL COMPOSITION CONTAINING THE SAME
CA2162497A1 (en) 1993-06-10 1994-12-22 Sheila Connelly Adenoviral vectors for treatment of hemophilia
SE504074C2 (en) 1993-07-05 1996-11-04 Pharmacia Ab Protein preparation for subcutaneous, intramuscular or intradermal administration
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
US5952198A (en) * 1995-05-04 1999-09-14 Bayer Corporation Production of recombinant Factor VIII in the presence of liposome-like substances of mixed composition
SE9503380D0 (en) 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
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
CN1179976C (en) * 2000-12-29 2004-12-15 中国科学院上海生物化学研究所 Production method and host cell for producing blood coagulation factor VIII
EP1572889B1 (en) 2001-10-05 2008-12-17 Expression Therapeutics, LLC Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
JP4810062B2 (en) 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 Sequence of adeno-associated virus (AAV) serotype 8
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
JP4644663B2 (en) 2003-06-03 2011-03-02 セル ジェネシス インコーポレイテッド Construction and method for enhanced expression of recombinant polypeptides from a single vector using peptide cleavage sites
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
CN101511373B (en) 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 Modified factor VIII and factor IX genes and vectors for gene therapy
KR101542752B1 (en) 2006-12-22 2015-08-10 체에스엘 베링 게엠베하 Modified coagulation factors with prolonged in vivo half-life
KR100959454B1 (en) 2007-12-10 2010-05-25 주식회사 동부하이텍 Semiconductor device and manufacturing method thereof
EP3586868B1 (en) 2008-04-22 2023-11-08 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
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
EA201291482A1 (en) 2010-07-09 2013-10-30 Байоджен Айдек Хемофилия Инк. CHEMERIC COAGULATION FACTORS
US20130017997A1 (en) * 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
WO2013016454A1 (en) 2011-07-25 2013-01-31 Biogen Idec Hemophilia Inc. Assays to monitor bleeding disorders
KR102212098B1 (en) * 2012-01-12 2021-02-03 바이오버라티브 테라퓨틱스 인크. Chimeric factor viii polypeptides and uses thereof
EA029560B1 (en) 2012-01-12 2018-04-30 Байоджен Эмэй Инк. Use of chimeric blood coagulation factor for reducing inhibitory immune response in a subject
MX369862B (en) * 2012-02-15 2019-11-25 Bioverativ Therapeutics Inc Factor viii compositions and methods of making and using same.
BR112014020325A2 (en) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia aav vector compositions and methods for gene transfer to cells, organs and tissues
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
GB201210357D0 (en) * 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
WO2014008172A2 (en) 2012-07-03 2014-01-09 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
WO2014064277A1 (en) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
PL2956477T5 (en) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Optimized factor viii gene
JP2016518321A (en) 2013-03-15 2016-06-23 バイエル・ヘルスケア・エルエルシーＢａｙｅｒ ＨｅａｌｔｈＣａｒｅ ＬＬＣ Recombinant factor VIII formulation
RS61305B1 (en) * 2013-06-24 2021-02-26 Xiao Weidong Mutant factor viii compositions and methods
RS61039B1 (en) 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vectors comprising a gene encoding factor viii
CN107427557B (en) 2014-08-13 2022-01-04 费城儿童医院 Improved expression modules for packaging and expressing variant factor VIII for treatment of hemophilia
CN114106146B (en) * 2015-02-06 2025-05-06 北卡罗来纳大学查珀尔希尔分校 Optimized human coagulation factor VIII gene expression cassette and its use
US11007280B2 (en) 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
EA202190827A1 (en) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед VIRAL VECTORS CODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A

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JP2021000091A (en) 2021-01-07
TWI777175B (en) 2022-09-11
US20170233455A1 (en) 2017-08-17
ZA201904814B (en) 2020-05-27
CL2018001299A1 (en) 2019-03-15
IL259302A (en) 2018-07-31
JP7631425B2 (en) 2025-02-18
US11254731B2 (en) 2022-02-22
EA038288B1 (en) 2021-08-05
EA202190827A1 (en) 2021-11-30
JP6768798B2 (en) 2020-10-14
CN108884145A (en) 2018-11-23
KR20230008256A (en) 2023-01-13
CN116479001A (en) 2023-07-25
IL281038B1 (en) 2023-10-01
NZ742557A (en) 2023-11-24
AU2020203760A1 (en) 2020-07-16
AU2016353353A1 (en) 2018-06-07
US20190202893A1 (en) 2019-07-04
BR112018009717A8 (en) 2019-02-26
ZA201803443B (en) 2019-03-27
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