AU2013276247B2 - Factor VIII sequences - Google Patents

Factor VIII sequences Download PDF

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Publication number
AU2013276247B2
AU2013276247B2 AU2013276247A AU2013276247A AU2013276247B2 AU 2013276247 B2 AU2013276247 B2 AU 2013276247B2 AU 2013276247 A AU2013276247 A AU 2013276247A AU 2013276247 A AU2013276247 A AU 2013276247A AU 2013276247 B2 AU2013276247 B2 AU 2013276247B2
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AU
Australia
Prior art keywords
sequence
nucleic acid
seq
acid molecule
factor viii
Prior art date
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Active
Application number
AU2013276247A
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English (en)
Other versions
AU2013276247A1 (en
Inventor
Andrew Davidoff
Jenny MCINTOSH
Amit Nathwani
Edward Tuddenham
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
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UCL Business Ltd
St Jude Childrens Research Hospital
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Publication of AU2013276247A1 publication Critical patent/AU2013276247A1/en
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Environmental Sciences (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Peptides Or Proteins (AREA)
AU2013276247A 2012-06-12 2013-06-12 Factor VIII sequences Active AU2013276247B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1210357.8 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 (2)

Publication Number Publication Date
AU2013276247A1 AU2013276247A1 (en) 2015-01-15
AU2013276247B2 true AU2013276247B2 (en) 2017-03-09

Family

ID=46605791

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2013276247A Active AU2013276247B2 (en) 2012-06-12 2013-06-12 Factor VIII sequences

Country Status (8)

Country Link
US (6) US9447168B2 (enExample)
EP (1) EP2859016B1 (enExample)
JP (1) JP6473416B2 (enExample)
AU (1) AU2013276247B2 (enExample)
CA (1) CA2875449A1 (enExample)
ES (1) ES2640586T3 (enExample)
GB (1) GB201210357D0 (enExample)
WO (1) WO2013186563A2 (enExample)

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GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
CA3229275A1 (en) 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
CN115094062A (zh) * 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
MX2018003702A (es) 2015-09-24 2018-04-30 Biomarin Pharm Inc Vectores del factor viii del virus adenoasociado, particulas viricas asociadas y formulaciones terapeuticas que las comprenden.
EA202190827A1 (ru) * 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
MX2018005969A (es) 2015-11-13 2018-11-29 Baxalta Inc Vectores virales que codifican variantes del factor fviii recombinantes con mayor expresión para la terapia génica de hemofilia a.
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
EP3790627A2 (en) 2018-05-09 2021-03-17 BioMarin Pharmaceutical Inc. Methods of treating phenylketonuria
JP2021523198A (ja) 2018-05-14 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド 幼若対象におけるａａｖベクターの安定的発現
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
KR20210034013A (ko) 2018-07-16 2021-03-29 박스알타 인코퍼레이티드 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법
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
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc 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
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
JP7733110B2 (ja) 2020-11-02 2025-09-02 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルスを濃縮するためのプロセス
WO2022264040A1 (en) 2021-06-14 2022-12-22 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
EP4463475A2 (en) 2022-01-14 2024-11-20 Anjarium Biosciences AG Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
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

Citations (1)

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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

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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
BRPI0109494B8 (pt) 2000-03-22 2021-05-25 Octagene Gmbh muteína de fator viii, sequência de dna, vetor, processo para produção da muteína de fator viii, composição farmacêutica e uso da muteína do fator viii
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Patent Citations (1)

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Also Published As

Publication number Publication date
US11419920B2 (en) 2022-08-23
EP2859016B1 (en) 2017-08-09
CA2875449A1 (en) 2013-12-19
US20250108093A1 (en) 2025-04-03
US10792336B2 (en) 2020-10-06
ES2640586T3 (es) 2017-11-03
JP6473416B2 (ja) 2019-02-20
WO2013186563A3 (en) 2014-02-13
US9447168B2 (en) 2016-09-20
US20170049859A1 (en) 2017-02-23
US20150158930A1 (en) 2015-06-11
EP2859016A2 (en) 2015-04-15
AU2013276247A1 (en) 2015-01-15
US10124041B2 (en) 2018-11-13
WO2013186563A2 (en) 2013-12-19
US20180311317A1 (en) 2018-11-01
US20200390866A1 (en) 2020-12-17
JP2015521465A (ja) 2015-07-30
GB201210357D0 (en) 2012-07-25
US20230241180A1 (en) 2023-08-03

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