FI3906066T3 - Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi - Google Patents

Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi Download PDF

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Publication number
FI3906066T3
FI3906066T3 FIEP20704633.5T FI20704633T FI3906066T3 FI 3906066 T3 FI3906066 T3 FI 3906066T3 FI 20704633 T FI20704633 T FI 20704633T FI 3906066 T3 FI3906066 T3 FI 3906066T3
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Finland
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sequence
taav
aav
itr
composition
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FIEP20704633.5T
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English (en)
Finnish (fi)
Inventor
Christine Livingston
Samuel Wadsworth
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Ultragenyx Pharmaceutical Inc
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (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)
FIEP20704633.5T 2019-01-04 2020-01-03 Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi FI3906066T3 (fi)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962788324P 2019-01-04 2019-01-04
US201962834830P 2019-04-16 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
FI3906066T3 true FI3906066T3 (fi) 2024-01-30

Family

ID=69528954

Family Applications (1)

Application Number Title Priority Date Filing Date
FIEP20704633.5T FI3906066T3 (fi) 2019-01-04 2020-01-03 Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi

Country Status (22)

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US (2) US12338450B2 (enExample)
EP (2) EP3906066B1 (enExample)
JP (2) JP7531495B2 (enExample)
KR (1) KR20210112339A (enExample)
CN (1) CN113518628B (enExample)
AU (1) AU2020204679A1 (enExample)
BR (1) BR112021013140A2 (enExample)
CA (1) CA3124880A1 (enExample)
CO (1) CO2021010088A2 (enExample)
DK (1) DK3906066T5 (enExample)
ES (1) ES2970216T3 (enExample)
FI (1) FI3906066T3 (enExample)
HR (1) HRP20231750T1 (enExample)
HU (1) HUE065782T2 (enExample)
IL (1) IL284554A (enExample)
MX (1) MX2021008135A (enExample)
PL (1) PL3906066T3 (enExample)
PT (1) PT3906066T (enExample)
SG (1) SG11202106855YA (enExample)
TW (1) TWI848038B (enExample)
WO (1) WO2020142653A1 (enExample)
ZA (1) ZA202104515B (enExample)

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SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
US12338450B2 (en) 2019-01-04 2025-06-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating Wilson disease
AU2020241888A1 (en) 2019-03-21 2021-09-30 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors
CN114072514A (zh) * 2019-05-14 2022-02-18 杜克大学 用于治疗atp酶介导的疾病的组合物和方法
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
AR123288A1 (es) 2020-08-19 2022-11-16 Stridebio Inc Vectores de virus adenoasociados para el tratamiento del síndrome de rett
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
KR20240114769A (ko) * 2021-12-10 2024-07-24 애반티바이오, 인크. 증진된 발현을 위해 선택된 코작 서열을 포함하는 조성물
US20250041453A1 (en) * 2021-12-10 2025-02-06 University Of Florida Research Foundation, Incorporated Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector
WO2024183769A1 (en) * 2023-03-06 2024-09-12 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
WO2025026198A1 (en) * 2023-07-28 2025-02-06 Lingyi Biotech Co., Ltd. Polynucleotides for the treatment of wilson disease

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Publication number Publication date
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
HUE065782T2 (hu) 2024-06-28
MX2021008135A (es) 2021-08-11
ZA202104515B (en) 2024-12-18
CN113518628B (zh) 2025-04-29
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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