ES2640586T3 - Secuencias del factor VIII - Google Patents

Secuencias del factor VIII Download PDF

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Publication number
ES2640586T3
ES2640586T3 ES13728823.9T ES13728823T ES2640586T3 ES 2640586 T3 ES2640586 T3 ES 2640586T3 ES 13728823 T ES13728823 T ES 13728823T ES 2640586 T3 ES2640586 T3 ES 2640586T3
Authority
ES
Spain
Prior art keywords
codop
hfviii
vector
aav
factor viii
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
ES13728823.9T
Other languages
English (en)
Spanish (es)
Inventor
Amit Nathwani
Jenny MCINTOSH
Edward Tuddenham
Andrew Davidoff
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
St Jude Childrens Research Hospital
Original Assignee
UCL Business Ltd
St Jude Childrens Research Hospital
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 UCL Business Ltd, St Jude Childrens Research Hospital filed Critical UCL Business Ltd
Application granted granted Critical
Publication of ES2640586T3 publication Critical patent/ES2640586T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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)
    • 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
    • A01K2267/01—Animal expressing industrially exogenous proteins
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Environmental Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Biotechnology (AREA)
  • Animal Husbandry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (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)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ES13728823.9T 2012-06-12 2013-06-12 Secuencias del factor VIII Active ES2640586T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB201210357 2012-06-12
GBGB1210357.8A GB201210357D0 (en) 2012-06-12 2012-06-12 Factor VIII sequences
PCT/GB2013/051551 WO2013186563A2 (en) 2012-06-12 2013-06-12 Factor viii sequences

Publications (1)

Publication Number Publication Date
ES2640586T3 true ES2640586T3 (es) 2017-11-03

Family

ID=46605791

Family Applications (1)

Application Number Title Priority Date Filing Date
ES13728823.9T Active ES2640586T3 (es) 2012-06-12 2013-06-12 Secuencias del factor VIII

Country Status (7)

Country Link
US (6) US9447168B2 (https=)
EP (1) EP2859016B1 (https=)
JP (1) JP6473416B2 (https=)
AU (1) AU2013276247B2 (https=)
ES (1) ES2640586T3 (https=)
GB (1) GB201210357D0 (https=)
WO (1) WO2013186563A2 (https=)

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GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
TW202446416A (zh) 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
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
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
TW202016298A (zh) 2018-05-14 2020-05-01 美商拜奧馬林製藥公司 Aav 載體於幼年受試者中之穩定表現
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
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
BR112021015776A2 (pt) * 2019-06-05 2021-11-09 Bayer Healthcare Llc Edição de gene para hemofilia a com expressão de fator viii melhorada
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
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
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
EP4355768A1 (en) 2021-06-14 2024-04-24 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
WO2024007978A1 (zh) 2022-07-07 2024-01-11 深圳新诺微环生物科技有限公司 一种连接肽、包含连接肽的凝血八因子蛋白或其变体及其用途
WO2024238591A2 (en) 2023-05-15 2024-11-21 Biomarin Pharmaceutical Inc. Methods of treating anti-aav seropositive hemophilia patients
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 (24)

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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
AU5689699A (en) 1998-08-24 2000-03-14 Uab Research Foundation Methods of producing high titer recombinant adeno-associated virus
US20090148906A1 (en) 1998-09-29 2009-06-11 Shire Human Genetic Therapies, Inc. A Delaware Corporation Optimized messenger rna
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US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
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WO2006036502A2 (en) 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
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
WO2007084487A2 (en) 2006-01-13 2007-07-26 Wayne State University Pericytes for use as stem cells
ES2535877T3 (es) 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
DK2173888T3 (en) 2007-07-26 2016-11-28 Uniqure Ip Bv Baculovirus vectors comprising repeating coding sequences WITH differential preferred codons
WO2009017533A1 (en) 2007-07-27 2009-02-05 Porteon Electric Vehicles, Inc. Modular light vehicle chassis
JP5739865B2 (ja) * 2009-03-24 2015-06-24 バイエル・ヘルスケア・エルエルシー 第ｖｉｉｉ因子変異体および使用の方法
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WO2011139629A2 (en) * 2010-04-26 2011-11-10 Biogen Idec Ma Inc. Light targeting molecules and uses thereof
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EP3044231B1 (en) 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii

Also Published As

Publication number Publication date
US20170049859A1 (en) 2017-02-23
WO2013186563A3 (en) 2014-02-13
AU2013276247A1 (en) 2015-01-15
US20250108093A1 (en) 2025-04-03
GB201210357D0 (en) 2012-07-25
US20180311317A1 (en) 2018-11-01
CA2875449A1 (en) 2013-12-19
US9447168B2 (en) 2016-09-20
JP2015521465A (ja) 2015-07-30
US20230241180A1 (en) 2023-08-03
AU2013276247B2 (en) 2017-03-09
US10124041B2 (en) 2018-11-13
US20150158930A1 (en) 2015-06-11
US20200390866A1 (en) 2020-12-17
US11419920B2 (en) 2022-08-23
JP6473416B2 (ja) 2019-02-20
EP2859016A2 (en) 2015-04-15
US10792336B2 (en) 2020-10-06
EP2859016B1 (en) 2017-08-09
WO2013186563A2 (en) 2013-12-19

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