JP7684212B2 - 第ｖｉｉｉ因子の発現による改善された臨床パラメーター - Google Patents

第ｖｉｉｉ因子の発現による改善された臨床パラメーター Download PDF

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Publication number
JP7684212B2
JP7684212B2 JP2021504376A JP2021504376A JP7684212B2 JP 7684212 B2 JP7684212 B2 JP 7684212B2 JP 2021504376 A JP2021504376 A JP 2021504376A JP 2021504376 A JP2021504376 A JP 2021504376A JP 7684212 B2 JP7684212 B2 JP 7684212B2
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aav
vector
seq
fviii
sequence
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JP2021533098A (ja
JPWO2020028830A5 (enExample
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エドワード・アール・コナー
ブリジット・イー・ライリー
ディディエ・ルイ
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Sangamo 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
    • 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
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—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
    • C12N2750/14071—Demonstrated in vivo effect
    • 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

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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)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021504376A 2018-08-03 2019-08-02 第ｖｉｉｉ因子の発現による改善された臨床パラメーター Active JP7684212B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862714553P 2018-08-03 2018-08-03
US62/714,553 2018-08-03
US201962826887P 2019-03-29 2019-03-29
US62/826,887 2019-03-29
US201962869445P 2019-07-01 2019-07-01
US62/869,445 2019-07-01
PCT/US2019/044946 WO2020028830A1 (en) 2018-08-03 2019-08-02 Improved clinical parameters by expression of factor viii

Publications (3)

Publication Number Publication Date
JP2021533098A JP2021533098A (ja) 2021-12-02
JPWO2020028830A5 JPWO2020028830A5 (enExample) 2022-08-09
JP7684212B2 true JP7684212B2 (ja) 2025-05-27

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JP2021504376A Active JP7684212B2 (ja) 2018-08-03 2019-08-02 第ｖｉｉｉ因子の発現による改善された臨床パラメーター

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Country Link
US (1) US12497632B2 (enExample)
EP (1) EP3829650A4 (enExample)
JP (1) JP7684212B2 (enExample)
KR (1) KR20210039428A (enExample)
CN (1) CN112533645A (enExample)
AU (1) AU2019314513A1 (enExample)
BR (1) BR112021001315A2 (enExample)
CA (1) CA3105382A1 (enExample)
IL (1) IL280402A (enExample)
MX (1) MX2021001375A (enExample)
WO (1) WO2020028830A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL303737A (en) 2020-12-18 2023-08-01 Sangamo Therapeutics Inc Improved pharmaceutical compositions containing adeno-associated viral vector

Citations (2)

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Publication number Priority date Publication date Assignee Title
WO2017053677A1 (en) 2015-09-24 2017-03-30 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
WO2017074526A1 (en) 2015-10-28 2017-05-04 Sangamo Biosciences, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof

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AU5588298A (en) 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
CA2392863A1 (en) 2002-07-08 2004-01-08 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw A high capacity recombinant adenoviral vector for treatment of hemophilia a
EP1675956B1 (en) 2003-10-21 2010-10-13 Merck Serono SA Minimal dna sequence acting as a chromatin insulator and its use in protein expression
JPWO2006043354A1 (ja) 2004-10-20 2008-05-22 独立行政法人放射線医学総合研究所 組込型の低線量放射線誘導性ベクター
CA2611465A1 (en) 2005-06-10 2007-02-22 Genentech, Inc. Improving antibody expression using vectors containing insulator elements
KR20100113478A (ko) 2007-11-01 2010-10-21 유니버시티 오브 로체스터 증가된 안정성을 갖는 재조합 인자 ⅴⅰⅰⅰ
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
WO2009134681A2 (en) * 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
KR102061557B1 (ko) 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
EP4144378A1 (en) * 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
RS59670B1 (sr) 2012-01-12 2020-01-31 Bioverativ Therapeutics Inc Himerni polipeptidi faktora viii i njihove upotrebe
EP3816281A1 (en) 2012-07-11 2021-05-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
WO2014043131A1 (en) 2012-09-14 2014-03-20 The Regents Of The University Of California Lentiviral vector for stem cell gene therapy of sickle cell disease
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
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MX388739B (es) * 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
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WO2017074526A1 (en) 2015-10-28 2017-05-04 Sangamo Biosciences, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof

Also Published As

Publication number Publication date
BR112021001315A2 (pt) 2021-04-27
US12497632B2 (en) 2025-12-16
KR20210039428A (ko) 2021-04-09
WO2020028830A1 (en) 2020-02-06
CN112533645A (zh) 2021-03-19
CA3105382A1 (en) 2020-02-06
EP3829650A4 (en) 2022-05-04
AU2019314513A1 (en) 2021-01-28
EP3829650A1 (en) 2021-06-09
JP2021533098A (ja) 2021-12-02
IL280402A (en) 2021-03-01
MX2021001375A (es) 2021-04-19
US20210171982A1 (en) 2021-06-10

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