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PL2420256T3 - Terapia genowa dla zaburzeń neurometabolicznych - Google Patents

Terapia genowa dla zaburzeń neurometabolicznych

Info

Publication number
PL2420256T3
PL2420256T3 PL11169633.2T PL11169633T PL2420256T3 PL 2420256 T3 PL2420256 T3 PL 2420256T3 PL 11169633 T PL11169633 T PL 11169633T PL 2420256 T3 PL2420256 T3 PL 2420256T3
Authority
PL
Poland
Prior art keywords
disorders
disease
nervous system
central nervous
gene therapy
Prior art date
Application number
PL11169633.2T
Other languages
English (en)
Inventor
Marco A. Passini
James Dodge
Gregory R Stewart
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed filed Critical
Publication of PL2420256T3 publication Critical patent/PL2420256T3/pl

Links

Classifications

    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • 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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates 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
    • 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
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • 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
    • C12N2750/14041Use 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
    • 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
PL11169633.2T 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych PL2420256T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US68580805P 2005-05-31 2005-05-31

Publications (1)

Publication Number Publication Date
PL2420256T3 true PL2420256T3 (pl) 2016-12-30

Family

ID=37308324

Family Applications (4)

Application Number Title Priority Date Filing Date
PL06759081T PL1879624T3 (pl) 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych
PL11169633.2T PL2420256T3 (pl) 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych
PL16162716T PL3058959T3 (pl) 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych
PL19153520T PL3520823T3 (pl) 2005-05-02 2006-05-02 Terapia genowa zaburzeń neurometabolicznych

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PL06759081T PL1879624T3 (pl) 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych

Family Applications After (2)

Application Number Title Priority Date Filing Date
PL16162716T PL3058959T3 (pl) 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych
PL19153520T PL3520823T3 (pl) 2005-05-02 2006-05-02 Terapia genowa zaburzeń neurometabolicznych

Country Status (13)

Country Link
US (2) US10632213B2 (pl)
EP (4) EP3520823B1 (pl)
JP (3) JP5829372B2 (pl)
CN (4) CN107007842A (pl)
AT (1) ATE525092T1 (pl)
AU (1) AU2006243776A1 (pl)
BR (1) BRPI0611379A2 (pl)
CA (2) CA2607173C (pl)
ES (1) ES2887076T3 (pl)
IL (2) IL187078A (pl)
PL (4) PL1879624T3 (pl)
PT (4) PT3058959T (pl)
WO (1) WO2006119458A1 (pl)

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Also Published As

Publication number Publication date
EP3520823B1 (en) 2021-06-30
IL187078A (en) 2015-06-30
CN104306986A (zh) 2015-01-28
US20080292593A1 (en) 2008-11-27
EP1879624B1 (en) 2011-09-21
CN107362371A (zh) 2017-11-21
EP3058959B1 (en) 2019-01-30
ES2887076T3 (es) 2021-12-21
BRPI0611379A2 (pt) 2010-08-31
JP2014037418A (ja) 2014-02-27
US11957765B2 (en) 2024-04-16
PT2420256T (pt) 2016-09-13
EP2420256A1 (en) 2012-02-22
US10632213B2 (en) 2020-04-28
CN107007842A (zh) 2017-08-04
US20200306387A1 (en) 2020-10-01
JP2016041706A (ja) 2016-03-31
CA2998603A1 (en) 2006-11-09
PL3058959T3 (pl) 2019-07-31
WO2006119458A9 (en) 2007-02-15
CN101212988A (zh) 2008-07-02
EP3058959A1 (en) 2016-08-24
EP3520823A1 (en) 2019-08-07
EP2420256B1 (en) 2016-06-22
WO2006119458A1 (en) 2006-11-09
US20140356327A9 (en) 2014-12-04
PT3520823T (pt) 2021-09-16
IL187078A0 (en) 2008-02-09
JP2008540445A (ja) 2008-11-20
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
AU2006243776A1 (en) 2006-11-09
PT3058959T (pt) 2019-05-24
IL239167A0 (en) 2015-07-30
PL3520823T3 (pl) 2021-11-22
EP1879624A4 (en) 2009-03-25
JP5829372B2 (ja) 2015-12-09
PL1879624T3 (pl) 2012-02-29
PT1879624E (pt) 2012-02-02
ATE525092T1 (de) 2011-10-15
JP6338560B2 (ja) 2018-06-06
EP1879624A1 (en) 2008-01-23
CA2607173A1 (en) 2006-11-09

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