JP6473416B2 - 第ｖｉｉｉ因子配列 - Google Patents

第ｖｉｉｉ因子配列 Download PDF

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Publication number
JP6473416B2
JP6473416B2 JP2015516684A JP2015516684A JP6473416B2 JP 6473416 B2 JP6473416 B2 JP 6473416B2 JP 2015516684 A JP2015516684 A JP 2015516684A JP 2015516684 A JP2015516684 A JP 2015516684A JP 6473416 B2 JP6473416 B2 JP 6473416B2
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Japan
Prior art keywords
nucleic acid
factor viii
vector
acid molecule
protein
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Expired - Fee Related
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JP2015516684A
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English (en)
Japanese (ja)
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JP2015521465A5 (OSRAM
JP2015521465A (ja
Inventor
アミット ナスワニ，
アミット ナスワニ，
ジェニー マッキントッシュ，
ジェニー マッキントッシュ，
エドワード タデナム，
エドワード タデナム，
アンドリュー ダビドフ，
アンドリュー ダビドフ，
Original Assignee
ユーシーエル ビジネス ピーエルシー
ユーシーエル ビジネス ピーエルシー
セント ジュード チルドレンズ リサーチ ホスピタル，インコーポレイティド
セント ジュード チルドレンズ リサーチ ホスピタル，インコーポレイティド
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Publication of JP2015521465A publication Critical patent/JP2015521465A/ja
Publication of JP2015521465A5 publication Critical patent/JP2015521465A5/ja
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    • 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)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (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)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
JP2015516684A 2012-06-12 2013-06-12 第ｖｉｉｉ因子配列 Expired - Fee Related JP6473416B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1210357.8A GB201210357D0 (en) 2012-06-12 2012-06-12 Factor VIII sequences
GB1210357.8 2012-06-12
PCT/GB2013/051551 WO2013186563A2 (en) 2012-06-12 2013-06-12 Factor viii sequences

Publications (3)

Publication Number Publication Date
JP2015521465A JP2015521465A (ja) 2015-07-30
JP2015521465A5 JP2015521465A5 (OSRAM) 2016-08-04
JP6473416B2 true JP6473416B2 (ja) 2019-02-20

Family

ID=46605791

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015516684A Expired - Fee Related JP6473416B2 (ja) 2012-06-12 2013-06-12 第ｖｉｉｉ因子配列

Country Status (8)

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

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* Cited by examiner, † Cited by third party
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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
ES3009565T3 (en) * 2015-04-16 2025-03-27 Univ Emory Recombinant promoters and vectors for protein expression in liver and use thereof
RU2020126034A (ru) 2015-09-24 2020-09-02 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
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
EA036944B1 (ru) * 2015-11-13 2021-01-19 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
US11584780B2 (en) 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
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
KR20210008491A (ko) 2018-05-14 2021-01-22 바이오마린 파머수티컬 인크. 청소년 대상체에서 aav 벡터의 안정한 발현
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
MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
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
MX2023005041A (es) 2020-11-02 2023-05-17 Biomarin Pharm Inc Proceso para enriquecer virus adenoasociado.
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
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
CN119233998A (zh) 2022-07-07 2024-12-31 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
WO2000011149A1 (en) 1998-08-24 2000-03-02 Uab Research Foundation Methods of producing high titer recombinant adeno-associated virus
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
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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
SI1266006T1 (sl) 2000-03-22 2006-06-30 Octagene Gmbh Proizvodnja rekombinantnih muteinov krvnega koagulacijskega faktorja VIII v humanih celicnih linijah
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
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
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ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
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
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CN101506369B (zh) 2006-06-21 2014-02-12 尤尼克尔生物制药股份有限公司 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
BRPI0814459B1 (pt) 2007-07-26 2023-01-24 Uniqure Ip B.V Método para produzir um vírion parvoviral recombinante em uma célula de inseto, e, construção de ácido nucleico
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GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
WO2011139629A2 (en) * 2010-04-26 2011-11-10 Biogen Idec Ma Inc. Light targeting molecules and uses thereof
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SI3044231T1 (sl) 2013-09-12 2020-12-31 Biomarin Pharmaceutical Inc. Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII

Also Published As

Publication number Publication date
WO2013186563A2 (en) 2013-12-19
US11419920B2 (en) 2022-08-23
US20230241180A1 (en) 2023-08-03
US10124041B2 (en) 2018-11-13
CA2875449A1 (en) 2013-12-19
EP2859016A2 (en) 2015-04-15
US10792336B2 (en) 2020-10-06
AU2013276247B2 (en) 2017-03-09
US20250108093A1 (en) 2025-04-03
ES2640586T3 (es) 2017-11-03
US9447168B2 (en) 2016-09-20
AU2013276247A1 (en) 2015-01-15
JP2015521465A (ja) 2015-07-30
US20170049859A1 (en) 2017-02-23
GB201210357D0 (en) 2012-07-25
US20180311317A1 (en) 2018-11-01
EP2859016B1 (en) 2017-08-09
US20200390866A1 (en) 2020-12-17
US20150158930A1 (en) 2015-06-11
WO2013186563A3 (en) 2014-02-13

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