JP7531495B2 - ウイルソン病を処置するための遺伝子治療構築物 - Google Patents

ウイルソン病を処置するための遺伝子治療構築物 Download PDF

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JP7531495B2
JP7531495B2 JP2021538754A JP2021538754A JP7531495B2 JP 7531495 B2 JP7531495 B2 JP 7531495B2 JP 2021538754 A JP2021538754 A JP 2021538754A JP 2021538754 A JP2021538754 A JP 2021538754A JP 7531495 B2 JP7531495 B2 JP 7531495B2
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JPWO2020142653A5 (enExample
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クリスティーン リビングストン，
サミュエル ウォズワース，
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ウルトラジェニックス ファーマシューティカル インコーポレイテッド
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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
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021538754A 2019-01-04 2020-01-03 ウイルソン病を処置するための遺伝子治療構築物 Active JP7531495B2 (ja)

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US201962788324P 2019-01-04 2019-01-04
US62/788,324 2019-01-04
US201962834830P 2019-04-16 2019-04-16
US62/834,830 2019-04-16
PCT/US2020/012131 WO2020142653A1 (en) 2019-01-04 2020-01-03 Gene therapy constructs for treating wilson disease

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CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN113694218A (zh) * 2021-08-30 2021-11-26 昆明理工大学 一种atp7b基因p992l突变的基因修复治疗载体
WO2023108130A2 (en) * 2021-12-10 2023-06-15 Aavantibio, Inc. Compositions comprising kozak sequences selected for enhanced expression
CA3242351A1 (en) * 2021-12-10 2023-06-15 University Of Florida Research Foundation, Incorporated Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector
CN120457199A (zh) * 2023-03-06 2025-08-08 凌意(杭州)生物科技有限公司 截短的atp7b及其用途
AU2024239180A1 (en) * 2023-03-22 2025-11-06 Skyline Therapeutics Limited Recombinant aav for the gene therapy of wilson disease
CN119220565A (zh) * 2023-06-28 2024-12-31 迈威(上海)生物科技股份有限公司 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用
CN120882865A (zh) * 2023-07-28 2025-10-31 凌意(杭州)生物科技有限公司 用于治疗威尔逊病的多核苷酸

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TW202043479A (zh) 2020-12-01
AU2020204679A1 (en) 2021-07-15
MX2021008135A (es) 2021-08-11
WO2020142653A1 (en) 2020-07-09
CN113518628A (zh) 2021-10-19
DK3906066T3 (da) 2024-02-05
EP3906066A1 (en) 2021-11-10
HUE065782T2 (hu) 2024-06-28
JP2022516283A (ja) 2022-02-25
PL3906066T3 (pl) 2024-09-02
IL284554A (en) 2021-08-31
KR20210112339A (ko) 2021-09-14
JP7746437B2 (ja) 2025-09-30
ES2970216T3 (es) 2024-05-27
US20220090131A1 (en) 2022-03-24
DK3906066T5 (da) 2024-08-05
HRP20231750T1 (hr) 2024-05-24
JP2024054426A (ja) 2024-04-16
ZA202104515B (en) 2024-12-18
CN113518628B (zh) 2025-04-29
EP4269579A3 (en) 2024-01-24
US20250333763A1 (en) 2025-10-30
BR112021013140A2 (pt) 2021-11-30
CA3124880A1 (en) 2020-07-09
FI3906066T3 (fi) 2024-01-30
EP3906066B1 (en) 2023-11-01
PT3906066T (pt) 2024-02-06
SG11202106855YA (en) 2021-07-29
EP4269579A2 (en) 2023-11-01
TWI848038B (zh) 2024-07-11
US12338450B2 (en) 2025-06-24
CO2021010088A2 (es) 2021-09-20

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