BRPI0611379A2 - terapia genética para distúrbios neurometabólicos - Google Patents

terapia genética para distúrbios neurometabólicos Download PDF

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Publication number
BRPI0611379A2
BRPI0611379A2 BRPI0611379-6A BRPI0611379A BRPI0611379A2 BR PI0611379 A2 BRPI0611379 A2 BR PI0611379A2 BR PI0611379 A BRPI0611379 A BR PI0611379A BR PI0611379 A2 BRPI0611379 A2 BR PI0611379A2
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BR
Brazil
Prior art keywords
aav
site
aav2
asm
disease
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BRPI0611379-6A
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English (en)
Portuguese (pt)
Inventor
Marco A Passini
James Dodge
Gregory R Stewart
Original Assignee
Genzyme Corp
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.)
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Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of BRPI0611379A2 publication Critical patent/BRPI0611379A2/pt

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    • 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
    • A61K48/0075Medicinal 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
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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

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  • 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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
BRPI0611379-6A 2005-05-02 2006-05-02 terapia genética para distúrbios neurometabólicos BRPI0611379A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US60/677,057 2005-05-02
US68580805P 2005-05-31 2005-05-31
US60/685,808 2005-05-31
PCT/US2006/017242 WO2006119458A1 (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Publications (1)

Publication Number Publication Date
BRPI0611379A2 true BRPI0611379A2 (pt) 2010-08-31

Family

ID=37308324

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0611379-6A BRPI0611379A2 (pt) 2005-05-02 2006-05-02 terapia genética para distúrbios neurometabólicos

Country Status (13)

Country Link
US (2) US10632213B2 (enExample)
EP (4) EP2420256B1 (enExample)
JP (3) JP5829372B2 (enExample)
CN (4) CN104306986A (enExample)
AT (1) ATE525092T1 (enExample)
AU (1) AU2006243776A1 (enExample)
BR (1) BRPI0611379A2 (enExample)
CA (2) CA2607173C (enExample)
ES (1) ES2887076T3 (enExample)
IL (2) IL187078A (enExample)
PL (4) PL3058959T3 (enExample)
PT (4) PT2420256T (enExample)
WO (1) WO2006119458A1 (enExample)

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US20200306387A1 (en) 2020-10-01
US20080292593A1 (en) 2008-11-27
JP6338560B2 (ja) 2018-06-06
EP2420256A1 (en) 2012-02-22
US11957765B2 (en) 2024-04-16
EP1879624A4 (en) 2009-03-25
JP2008540445A (ja) 2008-11-20
WO2006119458A1 (en) 2006-11-09
CN107362371A (zh) 2017-11-21
PT3058959T (pt) 2019-05-24
JP2016041706A (ja) 2016-03-31
JP2014037418A (ja) 2014-02-27
ATE525092T1 (de) 2011-10-15
JP5829372B2 (ja) 2015-12-09
US10632213B2 (en) 2020-04-28
PL1879624T3 (pl) 2012-02-29
PL3520823T3 (pl) 2021-11-22
PL2420256T3 (pl) 2016-12-30
CA2607173A1 (en) 2006-11-09
CN104306986A (zh) 2015-01-28
CN107007842A (zh) 2017-08-04
WO2006119458A9 (en) 2007-02-15
EP1879624A1 (en) 2008-01-23
IL239167A0 (en) 2015-07-30
IL187078A (en) 2015-06-30
CN101212988A (zh) 2008-07-02
EP3520823A1 (en) 2019-08-07
PT3520823T (pt) 2021-09-16
EP1879624B1 (en) 2011-09-21
PT2420256T (pt) 2016-09-13
IL187078A0 (en) 2008-02-09
EP2420256B1 (en) 2016-06-22
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
ES2887076T3 (es) 2021-12-21
EP3058959A1 (en) 2016-08-24
PT1879624E (pt) 2012-02-02
EP3058959B1 (en) 2019-01-30
CA2998603A1 (en) 2006-11-09
US20140356327A9 (en) 2014-12-04
PL3058959T3 (pl) 2019-07-31
EP3520823B1 (en) 2021-06-30
AU2006243776A1 (en) 2006-11-09

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