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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Japanese (ja)
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JP2015521465A5 (enExample
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)
  • 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)
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
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 (3)

Publication Number Publication Date
JP2015521465A JP2015521465A (ja) 2015-07-30
JP2015521465A5 JP2015521465A5 (enExample) 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 (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

Family Cites Families (24)

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Publication number Priority date Publication date Assignee Title
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
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US20090017533A1 (en) 1998-09-29 2009-01-15 Shire Human Genetic Therapies, Inc., A Delaware Corporation Optimized messenger rna
US6103526A (en) 1998-10-08 2000-08-15 Protein Sciences Corporation Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using
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
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
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
DK2438931T3 (da) 2004-09-22 2013-12-02 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
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
US20090175833A1 (en) 2006-01-13 2009-07-09 Paula Dore-Duffy Pericytes for use as stem cells
EP2037892B1 (en) 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
CA2655957C (en) 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
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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
GB201210357D0 (en) * 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
CA3178379A1 (en) 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors

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
WO2013186563A3 (en) 2014-02-13
US9447168B2 (en) 2016-09-20
US20170049859A1 (en) 2017-02-23
AU2013276247B2 (en) 2017-03-09
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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