CY1121380T1 - Γονιδιακη θεραπεια για τη νοσο νιεμann-pick τυπου a - Google Patents

Γονιδιακη θεραπεια για τη νοσο νιεμann-pick τυπου a

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Publication number
CY1121380T1
CY1121380T1 CY20181101265T CY181101265T CY1121380T1 CY 1121380 T1 CY1121380 T1 CY 1121380T1 CY 20181101265 T CY20181101265 T CY 20181101265T CY 181101265 T CY181101265 T CY 181101265T CY 1121380 T1 CY1121380 T1 CY 1121380T1
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CY
Cyprus
Prior art keywords
niemann
pick type
gene treatment
mammalian
polypeptide
Prior art date
Application number
CY20181101265T
Other languages
English (en)
Inventor
Marco A. Passini
Robin J. Ziegler
James Dodge
Lamya Shihabuddin
Seng H. Cheng
Original Assignee
Genzyme Corporation
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 by Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1121380T1 publication Critical patent/CY1121380T1/el

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    • CCHEMISTRY; METALLURGY
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • A61K48/0083Medicinal 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 administration regime
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive 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
    • 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
    • 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
    • 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
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
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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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/007Vectors comprising a special translation-regulating system cell or tissue specific
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00Screening for compounds of potential therapeutic value

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Η παρούσα αποκάλυψη αναφέρεται σε μεθόδους και συνθέσεις για πρόκληση ανοχής του εγκεφάλου ενός θηλαστικού στο εξωγενώς χορηγούμενο πολυπεπτίδιο όξινης σφιγγομυελινάσης χορηγώντας αρχικά μία αποτελεσματική ποσότητα ενός διαγονιδίου που κωδικοποιεί το πολυπεπτίδιο στον ηπατικό ιστό του θηλαστικού και στη συνέχεια χορηγώντας μια αποτελεσματική ποσότητα του διαγονιδίου στο κεντρικό νευρικό σύστημα (ΚΝΣ) του θηλαστικού.
CY20181101265T 2006-02-08 2018-11-28 Γονιδιακη θεραπεια για τη νοσο νιεμann-pick τυπου a CY1121380T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US77162806P 2006-02-08 2006-02-08
US77236006P 2006-02-09 2006-02-09
PCT/US2007/003388 WO2007092563A2 (en) 2006-02-08 2007-02-08 Gene therapy for niemann-pick disease type a

Publications (1)

Publication Number Publication Date
CY1121380T1 true CY1121380T1 (el) 2020-05-29

Family

ID=38345806

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20181101265T CY1121380T1 (el) 2006-02-08 2018-11-28 Γονιδιακη θεραπεια για τη νοσο νιεμann-pick τυπου a

Country Status (16)

Country Link
US (3) US20090117156A1 (el)
EP (2) EP3456331B1 (el)
JP (2) JP5706602B2 (el)
AR (1) AR059371A1 (el)
BR (1) BRPI0707590A2 (el)
CY (1) CY1121380T1 (el)
DK (1) DK1986661T3 (el)
ES (1) ES2700048T3 (el)
HU (1) HUE041928T2 (el)
IL (2) IL193165B (el)
LT (1) LT1986661T (el)
MX (3) MX360595B (el)
PL (1) PL1986661T3 (el)
PT (1) PT1986661T (el)
SI (1) SI1986661T1 (el)
WO (1) WO2007092563A2 (el)

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WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis
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Also Published As

Publication number Publication date
EP3456331B1 (en) 2021-05-26
US20200080066A1 (en) 2020-03-12
US20090117156A1 (en) 2009-05-07
WO2007092563A2 (en) 2007-08-16
JP5706602B2 (ja) 2015-04-22
EP1986661B1 (en) 2018-08-29
LT1986661T (lt) 2018-12-10
EP1986661A2 (en) 2008-11-05
EP1986661A4 (en) 2012-04-25
PL1986661T3 (pl) 2019-02-28
EP3456331A1 (en) 2019-03-20
US20230287366A1 (en) 2023-09-14
DK1986661T3 (da) 2019-01-02
MX2018013684A (es) 2021-06-15
JP2009526067A (ja) 2009-07-16
IL258587A (en) 2018-06-28
ES2700048T3 (es) 2019-02-13
JP5881641B2 (ja) 2016-03-09
SI1986661T1 (sl) 2018-12-31
BRPI0707590A2 (pt) 2011-05-10
JP2013166777A (ja) 2013-08-29
WO2007092563A3 (en) 2007-11-22
PT1986661T (pt) 2018-12-05
MX2018008355A (es) 2021-06-15
IL193165B (en) 2018-04-30
MX360595B (es) 2018-11-09
HUE041928T2 (hu) 2019-06-28
AR059371A1 (es) 2008-03-26

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