CA2607173C - Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease - Google Patents

Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease Download PDF

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Publication number
CA2607173C
CA2607173C CA2607173A CA2607173A CA2607173C CA 2607173 C CA2607173 C CA 2607173C CA 2607173 A CA2607173 A CA 2607173A CA 2607173 A CA2607173 A CA 2607173A CA 2607173 C CA2607173 C CA 2607173C
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composition according
composition
acid
alpha
administration
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Expired - Lifetime
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CA2607173A
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English (en)
French (fr)
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CA2607173A1 (en
Inventor
Marco A. Passini
James Dodge
Gregory R. Stewart
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Genzyme Corp
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Genzyme Corp
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates 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
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2750/14041—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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Physical Education & Sports Medicine (AREA)
CA2607173A 2005-05-02 2006-05-02 Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease Expired - Lifetime CA2607173C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA2998603A CA2998603C (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA2998603A Division CA2998603C (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Publications (2)

Publication Number Publication Date
CA2607173A1 CA2607173A1 (en) 2006-11-09
CA2607173C true CA2607173C (en) 2018-04-24

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
CA2607173A Expired - Lifetime CA2607173C (en) 2005-05-02 2006-05-02 Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease
CA2998603A Expired - Lifetime CA2998603C (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2998603A Expired - Lifetime CA2998603C (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Country Status (13)

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US (2) US10632213B2 (enExample)
EP (4) EP3520823B1 (enExample)
JP (3) JP5829372B2 (enExample)
CN (4) CN101212988A (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) PL3520823T3 (enExample)
PT (4) PT3520823T (enExample)
WO (1) WO2006119458A1 (enExample)

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PL3058959T3 (pl) 2019-07-31
PT3520823T (pt) 2021-09-16
EP3520823A1 (en) 2019-08-07
EP2420256A1 (en) 2012-02-22
JP5829372B2 (ja) 2015-12-09
PT3058959T (pt) 2019-05-24
BRPI0611379A2 (pt) 2010-08-31
EP3058959B1 (en) 2019-01-30
JP6338560B2 (ja) 2018-06-06
EP2420256B1 (en) 2016-06-22
CA2607173A1 (en) 2006-11-09
JP2014037418A (ja) 2014-02-27
JP2008540445A (ja) 2008-11-20
EP3520823B1 (en) 2021-06-30
IL187078A0 (en) 2008-02-09
WO2006119458A9 (en) 2007-02-15
PT1879624E (pt) 2012-02-02
PL3520823T3 (pl) 2021-11-22
US20140356327A9 (en) 2014-12-04
CA2998603C (en) 2019-07-23
US11957765B2 (en) 2024-04-16
AU2006243776A1 (en) 2006-11-09
EP1879624B1 (en) 2011-09-21
US20080292593A1 (en) 2008-11-27
CN107362371A (zh) 2017-11-21
WO2006119458A1 (en) 2006-11-09
PL2420256T3 (pl) 2016-12-30
US10632213B2 (en) 2020-04-28
CA2998603A1 (en) 2006-11-09
IL239167A0 (en) 2015-07-30
JP2016041706A (ja) 2016-03-31
EP1879624A1 (en) 2008-01-23
EP3058959A1 (en) 2016-08-24
PL1879624T3 (pl) 2012-02-29
CN107007842A (zh) 2017-08-04
EP1879624A4 (en) 2009-03-25
ATE525092T1 (de) 2011-10-15
ES2887076T3 (es) 2021-12-21
IL187078A (en) 2015-06-30
CN101212988A (zh) 2008-07-02
US20200306387A1 (en) 2020-10-01
PT2420256T (pt) 2016-09-13
CN104306986A (zh) 2015-01-28

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