JP7499174B2 - 第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法 - Google Patents

第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法 Download PDF

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JP7499174B2
JP7499174B2 JP2020505478A JP2020505478A JP7499174B2 JP 7499174 B2 JP7499174 B2 JP 7499174B2 JP 2020505478 A JP2020505478 A JP 2020505478A JP 2020505478 A JP2020505478 A JP 2020505478A JP 7499174 B2 JP7499174 B2 JP 7499174B2
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fviii
composition
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human
vector
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JP2020533276A5 (enExample
JP2020533276A (ja
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シャビエル アングエラ，
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Spark Therapeutics Inc
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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

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  • 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)
JP2020505478A 2017-08-01 2018-08-01 第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法 Active JP7499174B2 (ja)

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
US62/596,535 2017-12-08
US62/596,670 2017-12-08
PCT/US2018/044892 WO2019028192A1 (en) 2017-08-01 2018-08-01 METHODS OF GENE THERAPY TARGETING FACTOR VIII (FVIII)

Publications (3)

Publication Number Publication Date
JP2020533276A JP2020533276A (ja) 2020-11-19
JP2020533276A5 JP2020533276A5 (enExample) 2021-11-25
JP7499174B2 true JP7499174B2 (ja) 2024-06-13

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JP2020505478A Active JP7499174B2 (ja) 2017-08-01 2018-08-01 第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法

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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 (11)

* 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
MX2020007071A (es) 2018-01-05 2020-11-11 Platelet Biogenesis Inc Composiciones y metodos para producir megacariocitos.
WO2020006539A1 (en) 2018-06-29 2020-01-02 Platelet Biogenesis, Inc. Compositions for drug delivery and methods of use thereof
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
CA3133255A1 (en) * 2019-03-13 2020-09-17 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
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
WO2021084276A2 (en) * 2019-11-01 2021-05-06 Freeline Therapeutics Limited Factor viii construct
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017075619A1 (en) 2015-10-30 2017-05-04 Spark Therapeutics, Inc. Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

Family Cites Families (8)

* 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
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
WO2016025764A2 (en) * 2014-08-13 2016-02-18 The Children's Hospital Of Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
US10058624B2 (en) * 2015-04-16 2018-08-28 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
MA42934A (fr) * 2015-09-24 2018-08-01 Biomarin Pharm Inc Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci
MY189674A (en) * 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017075619A1 (en) 2015-10-30 2017-05-04 Spark Therapeutics, Inc. Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Faust, S. M. et al.，CpG-depleted adeno-associated virus vectors evade immune detection，The Journal of Clinical Investigation，2013年，Vol.123, No.7，p.2994-3001，doi:10.1172/JCI68205
綿野 亮太，水上浩明，血友病と遺伝子治療，小児内科，2017年07月，第49巻，第7号，p.1038-1043

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MX2020001402A (es) 2021-01-29
AU2018312565A1 (en) 2020-02-27
PE20200722A1 (es) 2020-07-21
KR20200066289A (ko) 2020-06-09
SG11202000650YA (en) 2020-02-27
KR20240160648A (ko) 2024-11-11
JP2020533276A (ja) 2020-11-19
MY208862A (en) 2025-06-04
EP3661541A1 (en) 2020-06-10
AU2024219336A1 (en) 2024-09-19
BR112020001979A2 (pt) 2020-08-18
CO2020002283A2 (es) 2020-04-24
CL2020000295A1 (es) 2020-12-04
IL272373A (en) 2020-03-31
CA3071519A1 (en) 2019-02-07
CN111163796A (zh) 2020-05-15
EP3661541A4 (en) 2021-09-01
PH12020500239A1 (en) 2021-01-11
US20200237930A1 (en) 2020-07-30
WO2019028192A1 (en) 2019-02-07
RU2020108209A (ru) 2021-09-02

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