ES2993915T3 - An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders - Google Patents

An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders Download PDF

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Publication number
ES2993915T3
ES2993915T3 ES15832128T ES15832128T ES2993915T3 ES 2993915 T3 ES2993915 T3 ES 2993915T3 ES 15832128 T ES15832128 T ES 15832128T ES 15832128 T ES15832128 T ES 15832128T ES 2993915 T3 ES2993915 T3 ES 2993915T3
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Prior art keywords
fviii
nucleic acid
aav
encoding
expression
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ES15832128T
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English (en)
Spanish (es)
Inventor
Denise Sabatino
Liron Elkouby
Katherine A High
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • 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
    • 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
    • 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
    • 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
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • 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
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ES15832128T 2014-08-13 2015-08-13 An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders Active ES2993915T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462036936P 2014-08-13 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

Publications (1)

Publication Number Publication Date
ES2993915T3 true ES2993915T3 (en) 2025-01-13

Family

ID=55304765

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15832128T Active ES2993915T3 (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

Country Status (12)

Country Link
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) CA2958141C (enExample)
ES (1) ES2993915T3 (enExample)
SA (1) SA517380883B1 (enExample)
SG (1) SG11201701033QA (enExample)
WO (1) WO2016025764A2 (enExample)
ZA (1) ZA201701039B (enExample)

Families Citing this family (21)

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ES2763551T3 (es) * 2015-04-16 2020-05-29 Univ Emory Promotores y vectores recombinantes para la expresión de proteínas en el hígado y uso de los mismos
CA2983593A1 (en) * 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
EP3361869B1 (en) 2015-10-14 2025-12-03 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
MY205187A (en) 2015-11-13 2024-10-04 Takeda Pharmaceuticals Co Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
MY198589A (en) 2015-11-13 2023-09-06 Takeda Pharmaceuticals Co Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
BR112019011115A2 (pt) 2016-12-02 2019-10-01 Bioverativ Therapeutics Inc. métodos para tratar artropatia hemofílica usando fatores de coagulação quiméricos
SG11202000650YA (en) * 2017-08-01 2020-02-27 Spark Therapeutics Inc Factor viii (fviii) gene therapy methods
WO2019210187A1 (en) * 2018-04-26 2019-10-31 The University Of North Carolina At Chapel Hill Methods and compositions for treatment of hemophilia
BR112020022164A2 (pt) 2018-05-18 2021-02-02 Bioverativ Therapeutics Inc. métodos de tratamento de hemofilia 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
TW202006141A (zh) 2018-07-16 2020-02-01 美商巴克斯歐塔公司 使用編碼具有增加表現的重組fviii變異體之病毒載體的a型血友病之基因療法
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
WO2022241041A2 (en) * 2021-05-11 2022-11-17 City Of Hope Adeno-associated virus compositions and methods of use thereof
JP7745307B2 (ja) * 2022-07-07 2025-09-29 深▲セン▼新▲諾▼▲微▼▲環▼生物科技有限公司 連結ペプチド、連結ペプチドを含む血液凝固ｖｉｉｉ因子タンパク質又はその変異体及びその用途
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

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP1866422B1 (en) * 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
US8198421B2 (en) * 2006-06-19 2012-06-12 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
US20120028900A1 (en) * 2006-06-30 2012-02-02 Kaufman Randal J Method of producing factor viii proteins by recombinant methods
CN101597616A (zh) * 2008-06-04 2009-12-09 上海同科生物科技有限公司 一种提高基因重组人凝血因子ⅷ表达量的方法
AU2013202564B2 (en) * 2008-06-24 2015-09-17 Csl Behring Gmbh Factor VIII, von Willebrand factor or complexes thereof with prolonged in vivo half-life
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
CA2776503C (en) * 2009-10-02 2020-07-28 The Children's Hospital Of Philadelphia Compositions and methods for enhancing coagulation factor viii function
US10023628B2 (en) * 2012-07-06 2018-07-17 Bioverativ Therapeutics Inc. Cell line expressing single chain factor VIII polypeptides and uses thereof
DK2911687T3 (da) * 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
HUE063051T2 (hu) * 2013-02-15 2023-12-28 Bioverativ Therapeutics Inc Optimalizált VIII. faktor gén

Also Published As

Publication number Publication date
CN107427557A (zh) 2017-12-01
JP2021003120A (ja) 2021-01-14
JP2018506261A (ja) 2018-03-08
CA3237630A1 (en) 2016-02-18
EP3180022A4 (en) 2019-02-20
ZA201701039B (en) 2021-08-25
JP7237903B2 (ja) 2023-03-13
NZ729107A (en) 2024-07-26
CN107427557B (zh) 2022-01-04
CA2958141A1 (en) 2016-02-18
KR20170084012A (ko) 2017-07-19
US11014975B2 (en) 2021-05-25
SA517380883B1 (ar) 2021-12-18
EP3180022B1 (en) 2024-07-03
EP3180022A2 (en) 2017-06-21
US20170233456A1 (en) 2017-08-17
CA2958141C (en) 2024-06-11
BR112017002781A2 (pt) 2017-12-19
AU2015301598B2 (en) 2020-07-16
AU2015301598A1 (en) 2017-03-23
KR102665348B1 (ko) 2024-05-16
WO2016025764A2 (en) 2016-02-18
WO2016025764A3 (en) 2016-04-07
SG11201701033QA (en) 2017-03-30

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