CN113518628B - 治疗威尔逊病的基因疗法构建体 - Google Patents

治疗威尔逊病的基因疗法构建体 Download PDF

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CN113518628B
CN113518628B CN202080007863.5A CN202080007863A CN113518628B CN 113518628 B CN113518628 B CN 113518628B CN 202080007863 A CN202080007863 A CN 202080007863A CN 113518628 B CN113518628 B CN 113518628B
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sequence
nucleic acid
raav
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CN113518628A (zh
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克里斯蒂娜·利文斯顿
塞缪尔·沃兹沃思
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Altraginx Pharmaceuticals
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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
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    • 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
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    • 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
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    • 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
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    • 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)
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  • Engineering & Computer Science (AREA)
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  • Medicinal Chemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CN202080007863.5A 2019-01-04 2020-01-03 治疗威尔逊病的基因疗法构建体 Active CN113518628B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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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CN113518628A CN113518628A (zh) 2021-10-19
CN113518628B true CN113518628B (zh) 2025-04-29

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KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
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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
KR20240114769A (ko) * 2021-12-10 2024-07-24 애반티바이오, 인크. 증진된 발현을 위해 선택된 코작 서열을 포함하는 조성물
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WO2025026198A1 (en) * 2023-07-28 2025-02-06 Lingyi Biotech Co., Ltd. Polynucleotides for the treatment of wilson disease

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US20220090131A1 (en) 2022-03-24
CO2021010088A2 (es) 2021-09-20
TWI848038B (zh) 2024-07-11
DK3906066T5 (da) 2024-08-05
EP4269579A2 (en) 2023-11-01
DK3906066T3 (da) 2024-02-05
FI3906066T3 (fi) 2024-01-30
HUE065782T2 (hu) 2024-06-28
MX2021008135A (es) 2021-08-11
ZA202104515B (en) 2024-12-18
JP7531495B2 (ja) 2024-08-09
EP3906066B1 (en) 2023-11-01
EP4269579A3 (en) 2024-01-24
PL3906066T3 (pl) 2024-09-02
CA3124880A1 (en) 2020-07-09
TW202043479A (zh) 2020-12-01
SG11202106855YA (en) 2021-07-29
WO2020142653A1 (en) 2020-07-09
EP3906066A1 (en) 2021-11-10
HRP20231750T1 (hr) 2024-05-24
US20250333763A1 (en) 2025-10-30
CN113518628A (zh) 2021-10-19
JP2024054426A (ja) 2024-04-16
JP2022516283A (ja) 2022-02-25
IL284554A (en) 2021-08-31
PT3906066T (pt) 2024-02-06
ES2970216T3 (es) 2024-05-27
AU2020204679A1 (en) 2021-07-15
KR20210112339A (ko) 2021-09-14
JP7746437B2 (ja) 2025-09-30
US12338450B2 (en) 2025-06-24
BR112021013140A2 (pt) 2021-11-30

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