KR20210112339A - 윌슨병을 치료하기 위한 유전자 요법 구축물 - Google Patents

윌슨병을 치료하기 위한 유전자 요법 구축물 Download PDF

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KR20210112339A
KR20210112339A KR1020217024152A KR20217024152A KR20210112339A KR 20210112339 A KR20210112339 A KR 20210112339A KR 1020217024152 A KR1020217024152 A KR 1020217024152A KR 20217024152 A KR20217024152 A KR 20217024152A KR 20210112339 A KR20210112339 A KR 20210112339A
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South Korea
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sequence
nucleic acid
raav
seq
atp7b
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KR1020217024152A
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Korean (ko)
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크리스틴 리빙스톤
사무엘 워즈워스
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울트라제닉스 파마수티컬 인코포레이티드
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Publication of KR20210112339A publication Critical patent/KR20210112339A/ko
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    • 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
    • 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
    • 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)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • 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)
KR1020217024152A 2019-01-04 2020-01-03 윌슨병을 치료하기 위한 유전자 요법 구축물 Pending KR20210112339A (ko)

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

Publications (1)

Publication Number Publication Date
KR20210112339A true KR20210112339A (ko) 2021-09-14

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ID=69528954

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217024152A Pending KR20210112339A (ko) 2019-01-04 2020-01-03 윌슨병을 치료하기 위한 유전자 요법 구축물

Country Status (22)

Country Link
US (2) US12338450B2 (https=)
EP (2) EP4269579B1 (https=)
JP (2) JP7531495B2 (https=)
KR (1) KR20210112339A (https=)
CN (1) CN113518628B (https=)
AU (1) AU2020204679B2 (https=)
BR (1) BR112021013140A2 (https=)
CA (1) CA3124880A1 (https=)
CO (1) CO2021010088A2 (https=)
DK (1) DK3906066T5 (https=)
ES (1) ES2970216T3 (https=)
FI (1) FI3906066T3 (https=)
HR (1) HRP20231750T1 (https=)
HU (1) HUE065782T2 (https=)
IL (1) IL284554A (https=)
MX (1) MX2021008135A (https=)
PL (1) PL3906066T3 (https=)
PT (1) PT3906066T (https=)
SG (1) SG11202106855YA (https=)
TW (1) TWI848038B (https=)
WO (1) WO2020142653A1 (https=)
ZA (1) ZA202104515B (https=)

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BR112019013245A2 (pt) 2016-12-30 2020-02-11 The Trustees Of The University Of Pennsylvania Terapia gênica para tratamento da doença de wilson
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
DK3906066T5 (da) 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3140049A1 (en) * 2019-05-14 2020-11-19 Duke University Compositions and methods for the treatment of atpase-mediated diseases
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. 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
CA3240197A1 (en) * 2021-12-10 2025-02-26 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
EP4677077A1 (en) * 2023-03-06 2026-01-14 Lingyi Biotech Co., Ltd. A truncated atp7b and the use thereof
WO2024193638A1 (en) * 2023-03-22 2024-09-26 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of wilson disease
WO2025002226A1 (zh) * 2023-06-28 2025-01-02 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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AU2002350039A1 (en) * 2001-10-29 2003-05-12 Renovis, Inc. Method for isolating cell-type specific mrnas
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
DE10156121A1 (de) 2001-11-16 2003-05-28 Wolfgang Stremmel Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
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EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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BR112019013245A2 (pt) 2016-12-30 2020-02-11 The Trustees Of The University Of Pennsylvania Terapia gênica para tratamento da doença de wilson
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Publication number Publication date
DK3906066T5 (da) 2024-08-05
MX2021008135A (es) 2021-08-11
CN113518628A (zh) 2021-10-19
EP4269579B1 (en) 2026-04-01
JP7746437B2 (ja) 2025-09-30
HUE065782T2 (hu) 2024-06-28
IL284554A (en) 2021-08-31
EP4269579A2 (en) 2023-11-01
JP2022516283A (ja) 2022-02-25
AU2020204679A1 (en) 2021-07-15
ZA202104515B (en) 2024-12-18
TW202043479A (zh) 2020-12-01
EP3906066B1 (en) 2023-11-01
HRP20231750T1 (hr) 2024-05-24
CO2021010088A2 (es) 2021-09-20
ES2970216T3 (es) 2024-05-27
US20220090131A1 (en) 2022-03-24
PL3906066T3 (pl) 2024-09-02
AU2020204679B2 (en) 2026-01-08
FI3906066T3 (fi) 2024-01-30
TWI848038B (zh) 2024-07-11
WO2020142653A1 (en) 2020-07-09
CN113518628B (zh) 2025-04-29
JP7531495B2 (ja) 2024-08-09
JP2024054426A (ja) 2024-04-16
US12338450B2 (en) 2025-06-24
CA3124880A1 (en) 2020-07-09
EP3906066A1 (en) 2021-11-10
SG11202106855YA (en) 2021-07-29
US20250333763A1 (en) 2025-10-30
DK3906066T3 (da) 2024-02-05
BR112021013140A2 (pt) 2021-11-30
PT3906066T (pt) 2024-02-06
EP4269579A3 (en) 2024-01-24

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