SA517380883B1 - Viii مجموعة تعبير متطورة لحزم والتعبير عن عامل متغير لعلاج اضطراب توقف نزيف الدم - Google Patents

Viii مجموعة تعبير متطورة لحزم والتعبير عن عامل متغير لعلاج اضطراب توقف نزيف الدم

Info

Publication number
SA517380883B1
SA517380883B1 SA517380883A SA517380883A SA517380883B1 SA 517380883 B1 SA517380883 B1 SA 517380883B1 SA 517380883 A SA517380883 A SA 517380883A SA 517380883 A SA517380883 A SA 517380883A SA 517380883 B1 SA517380883 B1 SA 517380883B1
Authority
SA
Saudi Arabia
Prior art keywords
factor viii
expression
packaging
treatment
variant factor
Prior art date
Application number
SA517380883A
Other languages
English (en)
Inventor
ساباتينو دينيس
ايه هاي كاثرين
الكوبي ليرون
Original Assignee
ذي شيلدرنـز هوسبيتال اوف فيلادلفيا
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ذي شيلدرنـز هوسبيتال اوف فيلادلفيا filed Critical ذي شيلدرنـز هوسبيتال اوف فيلادلفيا
Publication of SA517380883B1 publication Critical patent/SA517380883B1/ar

Links

Classifications

    • 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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (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)

Abstract

يتعلق الاختراع الحالي بالكشف عن متغيرات عامل VIII وطرق لاستخدامها. في نماذج معينة، يتم التعبير عن متغيرات عامل VIII بفعالية أكثر بواسطة الخلايا عن بروتينات عامل VIII من النوع غير المعالج ، ويتم إفرازها عند مستويات متزايدة من الخلايا عن بروتينات عامل VIII من النوع غير المعالج ، وتُظهر نشاط معزز عن بروتينات عامل VIII من النوع غير المعالج ويتم حزمها بفعالية أكثر في نواقل فيروسية. شكل6ب.
SA517380883A 2014-08-13 2017-02-12 Viii مجموعة تعبير متطورة لحزم والتعبير عن عامل متغير لعلاج اضطراب توقف نزيف الدم SA517380883B1 (ar)

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
SA517380883B1 true SA517380883B1 (ar) 2021-12-18

Family

ID=55304765

Family Applications (1)

Application Number Title Priority Date Filing Date
SA517380883A SA517380883B1 (ar) 2014-08-13 2017-02-12 Viii مجموعة تعبير متطورة لحزم والتعبير عن عامل متغير لعلاج اضطراب توقف نزيف الدم

Country Status (11)

Country Link
US (1) US11014975B2 (ar)
EP (1) EP3180022A4 (ar)
JP (2) JP2018506261A (ar)
CN (1) CN107427557B (ar)
AU (1) AU2015301598B2 (ar)
BR (1) BR112017002781A2 (ar)
CA (2) CA2958141A1 (ar)
SA (1) SA517380883B1 (ar)
SG (1) SG11201701033QA (ar)
WO (1) WO2016025764A2 (ar)
ZA (1) ZA201701039B (ar)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016168728A2 (en) * 2015-04-16 2016-10-20 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
EP3294309A4 (en) * 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF
AU2016338565B2 (en) 2015-10-14 2021-11-18 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
BR112018009732A8 (pt) 2015-11-13 2019-02-26 Baxalta GmbH ?polinucleotídeo, vetor de vírus adeno-associado, partícula de vírus adeno-associado, célula hospedeira, métodos para produzir uma partícula de vírus adeno-associado, para tratar hemofilia a, para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado?
TW202302627A (zh) 2015-11-13 2023-01-16 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變異體之病毒載體
MX2020001402A (es) * 2017-08-01 2021-01-29 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii).
SG11202010767SA (en) 2018-05-18 2020-11-27 Bioverativ Therapeutics Inc Methods of treating hemophilia 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
TW202039546A (zh) 2019-01-16 2020-11-01 美商巴克斯歐塔公司 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
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
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
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

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
EP3085389A1 (en) * 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP2037892B1 (en) * 2006-06-19 2015-03-18 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
EP2482841B2 (en) 2009-10-02 2022-09-28 The Children's Hospital Of Philadelphia Compositions and methods for enhancing coagulation factor viii function
EP2870250B2 (en) * 2012-07-06 2022-06-29 Bioverativ Therapeutics Inc. Cell line expressing single chain factor viii polypeptides and uses thereof
AU2013336601B2 (en) * 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
DK3889173T3 (da) * 2013-02-15 2023-10-09 Bioverativ Therapeutics Inc Optimeret faktor viii-gen

Also Published As

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

Similar Documents

Publication Publication Date Title
SA517380883B1 (ar) Viii مجموعة تعبير متطورة لحزم والتعبير عن عامل متغير لعلاج اضطراب توقف نزيف الدم
PH12018501055A1 (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
ZA201702632B (en) Compositions comprising recombinant bacillus cells and an insecticide
HK1249117A1 (zh) 優化的人類凝血因子viii基因表達盒及其用途
EA201891192A1 (ru) Средства и способы лечения миопатий, связанных с титином, и других титинопатий
PL3386009T3 (pl) Materiał aktywny elektrody dodatniej dla baterii akumulatorowej, elektroda dodatnia, dla baterii akumulatorowej, zawierającej to samo, i bateria akumulatorowa
SG10201810150UA (en) Compositions and methods for enhanced gene expression in cone cells
MX2021012014A (es) Vectores del factor viii del virus adeno-asociado.
EP3635108A4 (en) REINFORCEMENT AGENT FOR IMPROVED CELL TRANSFECTION AND / OR RAV VECTOR PRODUCTION
PH12016502248A1 (en) Adenoassociated virus vectors for the treatment of lysosomal storage disorders
MX2015012054A (es) Inhibidores covalentes de kras g12c.
CA2900779C (en) Compositions and methods for treating neurodegenerative diseases and cardiomyopathy
EA201390583A1 (ru) Композиции для улучшения состояния волос и соответствующие способы
EP2996725A4 (en) PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS AND METHODS FOR TREATING COLOR BLINDNESS AND OTHER PROBLEMS
CR20200609A (es) COMPOSICIONES Y MÉTODOS PARA DISMINUIR LA EXPRESIÓN DE TAU (Divisional 2018-334)
PH12016501689A1 (en) Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof
GB201513098D0 (en) Catalytically active protein aggregates and methods for producing the same
EP3998341A3 (en) Adenoviral vectors
HK1254181A1 (zh) 用於處理人角蛋白材料的裝置，特別是用於借助電流處理人角蛋白材料的裝置
MX2020012893A (es) Formulaciones de proteinas.
EP3236518A4 (en) Negative-electrode active material for electric device, and electric device using same
WO2014036524A3 (en) Genetically modified msc and therapeutic methods
IL274363B1 (en) Use of a specific anti-protein siRNA for the treatment of hemophilia
GB201619433D0 (en) Hair care agents containing casein hydrolysate for improving hair structure
WO2015143240A3 (en) Compositions and methods comprising 2-(acylamino)imidazoles