CO2021000468A2 - Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresión - Google Patents

Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresión

Info

Publication number
CO2021000468A2
CO2021000468A2 CONC2021/0000468A CO2021000468A CO2021000468A2 CO 2021000468 A2 CO2021000468 A2 CO 2021000468A2 CO 2021000468 A CO2021000468 A CO 2021000468A CO 2021000468 A2 CO2021000468 A2 CO 2021000468A2
Authority
CO
Colombia
Prior art keywords
hemophilia
gene therapy
viral vectors
vectors encoding
higher expression
Prior art date
Application number
CONC2021/0000468A
Other languages
English (en)
Inventor
Hanspeter Rottensteiner
Werner Hoellriegl
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 CO2021000468A2 publication Critical patent/CO2021000468A2/es

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    • 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
    • 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
    • C12N15/861—Adenoviral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • 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)
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
    • 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
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

RESUMEN La presente descripción proporciona, entre otros aspectos, polinucleótidos modificados con codones que codifican variantes del Factor VIII para su expresión en células de mamíferos. En algunas modalidades, la descripción también proporciona vectores de terapia génica de mamíferos y métodos para tratar la hemofilia A. En algunas modalidades, la presente descripción proporciona métodos para la dosificación de un paciente con hemofilia A con un polinucleótido, p. ej., un polinucleótido modificado con codones, que codifica un polipéptido del Factor VIII.
CONC2021/0000468A 2018-07-16 2021-01-19 Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresión CO2021000468A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862698680P 2018-07-16 2018-07-16
US201962867171P 2019-06-26 2019-06-26
PCT/US2019/041802 WO2020018419A1 (en) 2018-07-16 2019-07-15 Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression

Publications (1)

Publication Number Publication Date
CO2021000468A2 true CO2021000468A2 (es) 2021-04-08

Family

ID=67660451

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0000468A CO2021000468A2 (es) 2018-07-16 2021-01-19 Terapia génica de hemofilia a mediante el uso de vectores virales que codifican variantes del fviii recombinantes con mayor expresión

Country Status (12)

Country Link
US (2) US12091675B2 (es)
EP (1) EP3823985A1 (es)
JP (2) JP2021530524A (es)
KR (1) KR20210034013A (es)
CN (1) CN112449640A (es)
AU (1) AU2019306194A1 (es)
BR (1) BR112021000695A2 (es)
CA (1) CA3106590A1 (es)
CO (1) CO2021000468A2 (es)
MX (1) MX2021000582A (es)
TW (1) TW202006141A (es)
WO (1) WO2020018419A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2021000582A (es) 2018-07-16 2021-04-12 Baxalta Inc Terapia genica de hemofilia a mediante el uso de vectores virales que codifican variantes del factor viii (fviii) recombinantes con mayor expresion.
WO2021119357A2 (en) * 2019-12-12 2021-06-17 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
US20240269241A1 (en) * 2021-06-14 2024-08-15 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression

Family Cites Families (57)

* 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
JPH0788399B2 (ja) 1985-04-12 1995-09-27 ジェネティックス・インスチチュ−ト・インコ−ポレ−テッド 新規プロコアギュラント蛋白質
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
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
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
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
CA2162497A1 (en) 1993-06-10 1994-12-22 Sheila Connelly Adenoviral vectors for treatment of hemophilia
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
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
DE60230456D1 (de) 2001-10-05 2009-01-29 Expression Therapeutics Llc Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahren
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
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
CN104087591A (zh) 2006-06-19 2014-10-08 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子viii和因子ix基因和载体
WO2008069942A2 (en) 2006-12-05 2008-06-12 Biogen Idec Ma Inc. Novel methods of enhancing delivery of a gene therapy vector using steroids
EP2097096B1 (en) 2006-12-22 2017-05-31 CSL Behring GmbH Modified coagulation factors with prolonged in vivo half-life
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
JP5797551B2 (ja) 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
CN102482340B (zh) 2009-04-06 2015-05-13 诺沃—诺迪斯克有限公司 因子viii蛋白向血小板的靶向递送
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
LT2591006T (lt) 2010-07-09 2019-08-26 Bioverativ Therapeutics Inc. Apdorojamos viengrandės molekulės ir iš jų gauti polipeptidai
US20130017997A1 (en) 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
US10656167B2 (en) 2011-07-25 2020-05-19 Bioverativ Therapeutics Inc. Assays to monitor bleeding disorders
CN109111526A (zh) 2012-01-12 2019-01-01 比奥贝拉蒂治疗公司 嵌合因子viii多肽及其用途
SMT201800586T1 (it) 2012-01-12 2019-01-11 Bioverativ Therapeutics Inc Riduzione dell'immunogenicita' contro il fattore viii negli individui che si sottopongono alla terapia con fattore viii
SG10201609345QA (en) * 2012-02-07 2017-01-27 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
HRP20192314T1 (hr) 2012-02-15 2020-03-20 Bioverativ Therapeutics Inc. Pripravci faktora viii i postupci pripreme i njihove uporabe
ES2752191T3 (es) 2012-02-17 2020-04-03 Childrens Hospital Philadelphia Composiciones y métodos con vectores de AAV para la transferencia de genes a células, órganos y tejidos
HK1206612A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
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
EP3513802B1 (en) 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
CA2888931C (en) 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
FI2956477T4 (fi) 2013-02-15 2024-04-24 Bioverativ Therapeutics Inc Optimoitu tekijä viii:n geeni
SG10201803999UA (en) 2013-03-15 2018-06-28 Bayer Healthcare Llc Recombinant factor viii formulations
DK2796145T3 (da) * 2013-04-22 2018-01-29 Csl Ltd Et kovalent kompleks af von Willebrand-faktor og faktor VIII linket af en disulfidbro
SI3013855T1 (sl) 2013-06-24 2021-06-30 Xiao, Weidong Mutantne sestave faktorja VIII in metode
DK3044231T3 (da) 2013-09-12 2020-09-07 Biomarin Pharm Inc Aav-vektorer, der omfatter en genkodningsfaktor viii primær anmodning
EP3180022B1 (en) 2014-08-13 2024-07-03 The Children's Hospital of Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
KR102554850B1 (ko) 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
ES2764453T3 (es) 2015-03-17 2020-06-03 Univ Brussel Vrije Sistemas de expresión específica de hígado optimizados para FVIII y FIX
MA42934A (fr) * 2015-09-24 2018-08-01 Biomarin Pharm Inc Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci
AU2016353353B2 (en) 2015-11-13 2020-07-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
MX2021000582A (es) 2018-07-16 2021-04-12 Baxalta Inc Terapia genica de hemofilia a mediante el uso de vectores virales que codifican variantes del factor viii (fviii) recombinantes con mayor expresion.
JP7529286B2 (ja) 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用

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Publication number Publication date
KR20210034013A (ko) 2021-03-29
AU2019306194A1 (en) 2021-02-04
TW202006141A (zh) 2020-02-01
EP3823985A1 (en) 2021-05-26
US12091675B2 (en) 2024-09-17
BR112021000695A2 (pt) 2021-04-20
JP2021530524A (ja) 2021-11-11
US20250043310A1 (en) 2025-02-06
WO2020018419A1 (en) 2020-01-23
MX2021000582A (es) 2021-04-12
CA3106590A1 (en) 2020-01-23
US20220333135A1 (en) 2022-10-20
CN112449640A (zh) 2021-03-05
JP2024167251A (ja) 2024-12-03

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