JP7237364B2 - 遺伝子治療 - Google Patents

遺伝子治療 Download PDF

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Publication number
JP7237364B2
JP7237364B2 JP2019542703A JP2019542703A JP7237364B2 JP 7237364 B2 JP7237364 B2 JP 7237364B2 JP 2019542703 A JP2019542703 A JP 2019542703A JP 2019542703 A JP2019542703 A JP 2019542703A JP 7237364 B2 JP7237364 B2 JP 7237364B2
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sequence
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nucleic acid
mps
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JP2020506714A5 (enExample
JP2020506714A (ja
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ブライアン ビガー
エレーヌ グレイツ
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University of Manchester
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University of Manchester
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • 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
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C12N5/12—Fused cells, e.g. hybridomas
    • C12N5/16—Animal cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2019542703A 2017-02-07 2018-02-07 遺伝子治療 Active JP7237364B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1701968.8A GB201701968D0 (en) 2017-02-07 2017-02-07 Gene therapy
GB1701968.8 2017-02-07
PCT/GB2018/050347 WO2018146473A1 (en) 2017-02-07 2018-02-07 Gene therapy

Publications (3)

Publication Number Publication Date
JP2020506714A JP2020506714A (ja) 2020-03-05
JP2020506714A5 JP2020506714A5 (enExample) 2021-03-25
JP7237364B2 true JP7237364B2 (ja) 2023-03-13

Family

ID=58462471

Family Applications (1)

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JP2019542703A Active JP7237364B2 (ja) 2017-02-07 2018-02-07 遺伝子治療

Country Status (6)

Country Link
US (2) US11701390B2 (enExample)
EP (2) EP3960188B1 (enExample)
JP (1) JP7237364B2 (enExample)
ES (2) ES3035262T3 (enExample)
GB (1) GB201701968D0 (enExample)
WO (1) WO2018146473A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201701968D0 (en) * 2017-02-07 2017-03-22 Univ Manchester Gene therapy
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
WO2024096738A1 (en) 2022-11-01 2024-05-10 Erasmus University Medical Center Rotterdam Gene therapy constructs for metabolic disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007513188A (ja) 2003-12-04 2007-05-24 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ リソソーム蓄積症の処置のための組成物および方法
WO2016077356A2 (en) 2014-11-10 2016-05-19 Synageva Biopharma Corp. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003286870A1 (en) 2003-06-05 2005-01-04 Salk Institute For Biological Studies Targeting polypeptides to the central nervous system
US10227387B2 (en) 2011-05-18 2019-03-12 Children's Hospital Medical Center Targeted delivery of proteins across the blood-brain barrier
US20140335163A1 (en) 2011-12-01 2014-11-13 Angiochem Inc. Targeted iduronate-2-sulfatase compounds
GB201701968D0 (en) * 2017-02-07 2017-03-22 Univ Manchester Gene therapy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007513188A (ja) 2003-12-04 2007-05-24 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ リソソーム蓄積症の処置のための組成物および方法
WO2016077356A2 (en) 2014-11-10 2016-05-19 Synageva Biopharma Corp. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MOLECULAR THERAPY，2014年12月，VOL:22, NR:12，PAGE(S):2028 - 2037，http://dx.doi.org/10.1038/mt.2014.152
The J of Neuroscience, 2014, Vol.34, No.9, pp.3122-3129

Also Published As

Publication number Publication date
GB201701968D0 (en) 2017-03-22
US11701390B2 (en) 2023-07-18
WO2018146473A1 (en) 2018-08-16
EP3579849B1 (en) 2021-09-08
EP3579849A1 (en) 2019-12-18
JP2020506714A (ja) 2020-03-05
EP3960188A1 (en) 2022-03-02
US20200000854A1 (en) 2020-01-02
EP3960188B1 (en) 2025-04-02
ES2895263T3 (es) 2022-02-18
ES3035262T3 (en) 2025-09-01
US20230390340A1 (en) 2023-12-07

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