CN107427557B - 用于包装和表达变体因子viii以治疗血友病的改良表达组件 - Google Patents

用于包装和表达变体因子viii以治疗血友病的改良表达组件 Download PDF

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CN107427557B
CN107427557B CN201580055574.1A CN201580055574A CN107427557B CN 107427557 B CN107427557 B CN 107427557B CN 201580055574 A CN201580055574 A CN 201580055574A CN 107427557 B CN107427557 B CN 107427557B
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fviii
nucleic acid
aav
expression
vector
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CN107427557A (zh
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D·萨巴蒂诺
K·A·海
L·埃尔库比
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Childrens Hospital of Philadelphia CHOP
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    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • 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
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Diabetes (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201580055574.1A 2014-08-13 2015-08-13 用于包装和表达变体因子viii以治疗血友病的改良表达组件 Active CN107427557B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462036936P 2014-08-13 2014-08-13
US62/036,936 2014-08-13
PCT/US2015/045142 WO2016025764A2 (en) 2014-08-13 2015-08-13 An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders

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CN107427557A CN107427557A (zh) 2017-12-01
CN107427557B true CN107427557B (zh) 2022-01-04

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US (1) US11014975B2 (enExample)
EP (1) EP3180022B1 (enExample)
JP (2) JP2018506261A (enExample)
KR (1) KR102665348B1 (enExample)
CN (1) CN107427557B (enExample)
AU (1) AU2015301598B2 (enExample)
CA (2) CA3237630A1 (enExample)
ES (1) ES2993915T3 (enExample)
SA (1) SA517380883B1 (enExample)
SG (1) SG11201701033QA (enExample)
WO (1) WO2016025764A2 (enExample)
ZA (1) ZA201701039B (enExample)

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ES3009565T3 (en) * 2015-04-16 2025-03-27 Univ Emory Recombinant promoters and vectors for protein expression in liver and use thereof
JP6929230B2 (ja) * 2015-05-14 2021-09-01 セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド スペーサーを含む核酸分子およびその使用の方法
CA3001594A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
EA036944B1 (ru) 2015-11-13 2021-01-19 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
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
IL308416B2 (en) 2016-12-02 2025-08-01 Bioverativ Therapeutics Inc Methods of treating hemophilic arthropathy using chimeric clotting factors
MY208862A (en) * 2017-08-01 2025-06-04 Spark Therapeutics Inc Factor viii (fviii) gene therapy methods
EP3810647A4 (en) * 2018-04-26 2022-08-17 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR THE TREATMENT OF HEMOPHILIA
MA52630B1 (fr) 2018-05-18 2025-07-31 Bioverativ Therapeutics Inc. Procédés de traitement de l'hémophilie a
CN108795986A (zh) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 一种a型血友病慢病毒载体、慢病毒及其制备方法和应用
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
EP3736286A1 (en) * 2019-05-09 2020-11-11 Biotest AG Single chain factor viii molecule
CN111218446B (zh) * 2019-12-25 2023-03-28 劲帆生物医药科技（武汉）有限公司 一种肝脏特异性启动子及其应用
US20250101465A1 (en) * 2021-05-11 2025-03-27 City Of Hope Adeno-associated virus compositions 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
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
CN115873099B (zh) * 2022-12-30 2023-10-20 福因医药科技(武汉)有限公司 一种改造的重组凝血因子viii及其应用
CN116715752B (zh) * 2023-06-16 2024-11-15 苏州诺洁贝生物技术有限公司 凝血因子fviii蛋白变体、表达载体和用途

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US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
US6924365B1 (en) * 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
ES2428218T3 (es) * 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
CN101511373B (zh) * 2006-06-19 2015-12-09 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
US20120028900A1 (en) * 2006-06-30 2012-02-02 Kaufman Randal J Method of producing factor viii proteins by recombinant methods
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WO2016025764A2 (en) 2016-02-18
WO2016025764A3 (en) 2016-04-07
JP2021003120A (ja) 2021-01-14
AU2015301598A1 (en) 2017-03-23
BR112017002781A2 (pt) 2017-12-19
KR102665348B1 (ko) 2024-05-16
US11014975B2 (en) 2021-05-25
JP7237903B2 (ja) 2023-03-13
ES2993915T3 (en) 2025-01-13
NZ729107A (en) 2024-07-26
CA2958141C (en) 2024-06-11
US20170233456A1 (en) 2017-08-17
JP2018506261A (ja) 2018-03-08
EP3180022A4 (en) 2019-02-20
EP3180022A2 (en) 2017-06-21
KR20170084012A (ko) 2017-07-19
SG11201701033QA (en) 2017-03-30
CN107427557A (zh) 2017-12-01
CA3237630A1 (en) 2016-02-18
EP3180022B1 (en) 2024-07-03
AU2015301598B2 (en) 2020-07-16
CA2958141A1 (en) 2016-02-18
SA517380883B1 (ar) 2021-12-18
ZA201701039B (en) 2021-08-25

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