PL3233130T3 - Konstrukty kwasów nukleinowych i wektory do terapii genowej do zastosowania w leczeniu choroby wilsona - Google Patents

Konstrukty kwasów nukleinowych i wektory do terapii genowej do zastosowania w leczeniu choroby wilsona

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Publication number
PL3233130T3
PL3233130T3 PL15817240T PL15817240T PL3233130T3 PL 3233130 T3 PL3233130 T3 PL 3233130T3 PL 15817240 T PL15817240 T PL 15817240T PL 15817240 T PL15817240 T PL 15817240T PL 3233130 T3 PL3233130 T3 PL 3233130T3
Authority
PL
Poland
Prior art keywords
treatment
nucleic acid
gene therapy
acid constructs
therapy vectors
Prior art date
Application number
PL15817240T
Other languages
English (en)
Inventor
Oihana MURILLO SAUCA
Gloria GONZÁLEZ ASEGUINOLAZA
Rubén HERNÁNDEZ ALCOCEBA
Original Assignee
Fundacion Para La Investigacion Medica Aplicada
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Filing date
Publication date
Application filed by Fundacion Para La Investigacion Medica Aplicada filed Critical Fundacion Para La Investigacion Medica Aplicada
Publication of PL3233130T3 publication Critical patent/PL3233130T3/pl

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    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/02—Antidotes
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL15817240T 2014-12-17 2015-12-17 Konstrukty kwasów nukleinowych i wektory do terapii genowej do zastosowania w leczeniu choroby wilsona PL3233130T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14382531 2014-12-17
PCT/EP2015/080357 WO2016097219A1 (en) 2014-12-17 2015-12-17 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
EP15817240.3A EP3233130B1 (en) 2014-12-17 2015-12-17 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease

Publications (1)

Publication Number Publication Date
PL3233130T3 true PL3233130T3 (pl) 2021-11-22

Family

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Family Applications (1)

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PL15817240T PL3233130T3 (pl) 2014-12-17 2015-12-17 Konstrukty kwasów nukleinowych i wektory do terapii genowej do zastosowania w leczeniu choroby wilsona

Country Status (18)

Country Link
US (2) US11147887B2 (pl)
EP (2) EP3799889A1 (pl)
JP (1) JP6865167B2 (pl)
KR (1) KR102526616B1 (pl)
CN (1) CN107208105A (pl)
AU (1) AU2015367385B2 (pl)
CA (1) CA2985624C (pl)
DK (1) DK3233130T3 (pl)
ES (1) ES2876412T3 (pl)
HU (1) HUE055002T2 (pl)
IL (1) IL252917B2 (pl)
MX (1) MX387165B (pl)
PL (1) PL3233130T3 (pl)
PT (1) PT3233130T (pl)
RU (1) RU2745567C2 (pl)
SG (1) SG11201704690TA (pl)
SI (1) SI3233130T1 (pl)
WO (1) WO2016097219A1 (pl)

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EP3880809A4 (en) * 2018-11-16 2023-01-11 Encoded Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF WILSON'S DISEASE
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CN111088285B (zh) * 2019-08-15 2021-12-28 北京锦篮基因科技有限公司 携带atp7b基因表达框及变异体的aav载体及应用
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US20230265456A1 (en) * 2020-08-10 2023-08-24 Fundacion Para La Investigacion Medica Aplicada Gene therapy vector expressing cyp27a1 for the treatment of cerebrotendinous xanthomatosis
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US20240254509A1 (en) 2021-03-04 2024-08-01 Research Institute At Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
US20240307553A1 (en) * 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
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
WO2023038909A1 (en) * 2021-09-07 2023-03-16 Alexion Pharmaceuticals, Inc. Methods of treating copper metabolism-associated diseases or disorders
TW202323528A (zh) * 2021-10-18 2023-06-16 美商邏輯生物療法公司 用於治療威爾森氏病之基因療法
US20250320509A1 (en) * 2022-05-23 2025-10-16 The Johns Hopkins University Compositions and methods for treating wilson's disease
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Also Published As

Publication number Publication date
MX2017007965A (es) 2018-01-18
SG11201704690TA (en) 2017-07-28
AU2015367385B2 (en) 2021-05-27
US11147887B2 (en) 2021-10-19
JP6865167B2 (ja) 2021-04-28
JP2018500904A (ja) 2018-01-18
RU2017125234A (ru) 2019-01-17
BR112017012895A2 (pt) 2018-02-06
US20170348435A1 (en) 2017-12-07
IL252917B2 (en) 2023-10-01
ES2876412T3 (es) 2021-11-12
EP3233130A1 (en) 2017-10-25
US20220008559A1 (en) 2022-01-13
SI3233130T1 (sl) 2021-08-31
RU2017125234A3 (pl) 2019-07-25
HUE055002T2 (hu) 2021-10-28
CN107208105A (zh) 2017-09-26
PT3233130T (pt) 2021-06-24
EP3799889A1 (en) 2021-04-07
RU2745567C2 (ru) 2021-03-29
CA2985624A1 (en) 2016-06-23
MX387165B (es) 2025-03-18
IL252917A0 (en) 2017-08-31
KR102526616B1 (ko) 2023-04-27
KR20170108951A (ko) 2017-09-27
CA2985624C (en) 2023-06-13
EP3233130B1 (en) 2021-03-31
DK3233130T3 (da) 2021-06-21
AU2015367385A1 (en) 2017-06-29
IL252917B1 (en) 2023-06-01
WO2016097219A1 (en) 2016-06-23

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