AU2018312565A1 - Factor VIII (FVIII) gene therapy methods - Google Patents

Factor VIII (FVIII) gene therapy methods Download PDF

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Publication number
AU2018312565A1
AU2018312565A1 AU2018312565A AU2018312565A AU2018312565A1 AU 2018312565 A1 AU2018312565 A1 AU 2018312565A1 AU 2018312565 A AU2018312565 A AU 2018312565A AU 2018312565 A AU2018312565 A AU 2018312565A AU 2018312565 A1 AU2018312565 A1 AU 2018312565A1
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AU
Australia
Prior art keywords
human
fviii
raav vector
hfviii
bdd
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2018312565A
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English (en)
Inventor
Xavier ANGUELA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Spark Therapeutics Inc
Original Assignee
Spark Therapeutics Inc
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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of AU2018312565A1 publication Critical patent/AU2018312565A1/en
Priority to AU2024219336A priority Critical patent/AU2024219336A1/en
Abandoned legal-status Critical Current

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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
    • 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
    • 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)
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2018312565A 2017-08-01 2018-08-01 Factor VIII (FVIII) gene therapy methods Abandoned AU2018312565A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024219336A AU2024219336A1 (en) 2017-08-01 2024-09-03 Factor viii (fviii) gene therapy methods

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201762540053P 2017-08-01 2017-08-01
US62/540,053 2017-08-01
US201762583890P 2017-11-09 2017-11-09
US62/583,890 2017-11-09
US201762596535P 2017-12-08 2017-12-08
US201762596670P 2017-12-08 2017-12-08
US62/596,670 2017-12-08
US62/596,535 2017-12-08
PCT/US2018/044892 WO2019028192A1 (en) 2017-08-01 2018-08-01 METHODS OF GENE THERAPY TARGETING FACTOR VIII (FVIII)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024219336A Division AU2024219336A1 (en) 2017-08-01 2024-09-03 Factor viii (fviii) gene therapy methods

Publications (1)

Publication Number Publication Date
AU2018312565A1 true AU2018312565A1 (en) 2020-02-27

Family

ID=65234171

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2018312565A Abandoned AU2018312565A1 (en) 2017-08-01 2018-08-01 Factor VIII (FVIII) gene therapy methods
AU2024219336A Pending AU2024219336A1 (en) 2017-08-01 2024-09-03 Factor viii (fviii) gene therapy methods

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024219336A Pending AU2024219336A1 (en) 2017-08-01 2024-09-03 Factor viii (fviii) gene therapy methods

Country Status (17)

Country Link
US (1) US20200237930A1 (enExample)
EP (1) EP3661541A4 (enExample)
JP (1) JP7499174B2 (enExample)
KR (2) KR20200066289A (enExample)
CN (1) CN111163796A (enExample)
AU (2) AU2018312565A1 (enExample)
BR (1) BR112020001979A2 (enExample)
CA (1) CA3071519A1 (enExample)
CL (1) CL2020000295A1 (enExample)
CO (1) CO2020002283A2 (enExample)
IL (1) IL272373A (enExample)
MX (1) MX2020001402A (enExample)
MY (1) MY208862A (enExample)
PE (1) PE20200722A1 (enExample)
PH (1) PH12020500239A1 (enExample)
SG (1) SG11202000650YA (enExample)
WO (1) WO2019028192A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
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
JP2021509812A (ja) 2018-01-05 2021-04-08 プレートレット バイオジェネシス， インコーポレイテッド 巨核球を産生するための組成物および方法
WO2020006539A1 (en) 2018-06-29 2020-01-02 Platelet Biogenesis, Inc. Compositions for drug delivery and methods of use thereof
AU2019314513A1 (en) * 2018-08-03 2021-01-28 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
EP3923992A1 (en) 2019-02-15 2021-12-22 CRISPR Therapeutics AG Gene editing for hemophilia a with improved factor viii expression
MX2021011039A (es) * 2019-03-13 2021-12-15 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con fviii.
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
JP2023501262A (ja) * 2019-11-01 2023-01-18 フリーライン セラピューティクス リミテッド 転写調節エレメント
WO2021113800A1 (en) * 2019-12-06 2021-06-10 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor viii function
CN114989307B (zh) * 2022-05-11 2023-08-01 华兰生物工程股份有限公司 一种重组人凝血因子Ⅷ-Fc融合蛋白及制备方法
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 (9)

* Cited by examiner, † Cited by third party
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
EP1224312A1 (en) * 1999-10-12 2002-07-24 The University of North Carolina at Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
AU2014318890B2 (en) * 2013-09-12 2019-03-07 Biomarin Pharmaceutical Inc. Adeno-associated virus Factor VIII vectors
ES2993915T3 (en) * 2014-08-13 2025-01-13 Childrens Hospital Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
CN108093639B (zh) * 2015-04-16 2022-07-19 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
TWI841908B (zh) * 2015-09-24 2024-05-11 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
AU2016343887B2 (en) * 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes 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
US20190144524A1 (en) * 2016-01-14 2019-05-16 The Children's Hospital Of Philadelphia Factor viii variants, nucleic acid sequences, and methods and uses for treatment of hemostasis disorders

Also Published As

Publication number Publication date
EP3661541A4 (en) 2021-09-01
RU2020108209A (ru) 2021-09-02
KR20240160648A (ko) 2024-11-11
US20200237930A1 (en) 2020-07-30
EP3661541A1 (en) 2020-06-10
BR112020001979A2 (pt) 2020-08-18
WO2019028192A1 (en) 2019-02-07
PH12020500239A1 (en) 2021-01-11
MY208862A (en) 2025-06-04
MX2020001402A (es) 2021-01-29
CO2020002283A2 (es) 2020-04-24
AU2024219336A1 (en) 2024-09-19
CN111163796A (zh) 2020-05-15
IL272373A (en) 2020-03-31
CA3071519A1 (en) 2019-02-07
PE20200722A1 (es) 2020-07-21
JP7499174B2 (ja) 2024-06-13
JP2020533276A (ja) 2020-11-19
KR20200066289A (ko) 2020-06-09
CL2020000295A1 (es) 2020-12-04
SG11202000650YA (en) 2020-02-27

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MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted