HRP20171165T1 - Genska terapija za lizosomske bolesti nakupljanja - Google Patents

Genska terapija za lizosomske bolesti nakupljanja Download PDF

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Publication number
HRP20171165T1
HRP20171165T1 HRP20171165TT HRP20171165T HRP20171165T1 HR P20171165 T1 HRP20171165 T1 HR P20171165T1 HR P20171165T T HRP20171165T T HR P20171165TT HR P20171165 T HRP20171165 T HR P20171165T HR P20171165 T1 HRP20171165 T1 HR P20171165T1
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Croatia
Prior art keywords
aav vector
recombinant aav
use according
vector
brain
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HRP20171165TT
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English (en)
Inventor
James Dodge
Seng H. Cheng
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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.)
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Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of HRP20171165T1 publication Critical patent/HRP20171165T1/hr

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    • 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
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • 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
    • 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
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

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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)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)

Claims (6)

1. Rekombinantni AAV vektor koji sadrži transgen kodira kiselinu sfingomijelinazu za upotrebu u postupku liječenja Niemann–Pickove bolesti tip A ili Niemann–Pickove bolesti tip B kod subjekta, naznačen time da navedeni rekombinantni AAV vektor je AAV5 vektor i pri čemu postupak sadrži davanje vektora u barem jednu ventrikulu mozga od subjekta, pri čemu se navedeni transgen eksprimira i eksprimirani proizvod proteina se dovodi u središnji živčani sustav, te proizvoljno, u visceralne organe.
2. Rekombinantni AAV vektor za uporabu prema zahtjevu 1, naznačen time da navedeni rekombinantni AAV vektor je AAV vektor koji sadrži AAV5 ovojnicu i AAV2 genom.
3. Rekombinantni AAV vektor za uporabu prema zahtjevu 1 ili zahtjevu 2, naznačen time da postupak sadrži davanje vektora izravnim ubrizgavanjem u: (i) ventrikulu mozga; (ii) lateralnu ventrikulu mozga; ili (iii) četvrtu ventrikulu mozga.
4. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 3, naznačen time da subjekt je sisavac.
5. Rekombinantni AAV vektor za uporabu prema zahtjevu 4, naznačen time da subjekt je ljudski pacijent.
6. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 5, naznačen time da navedeni postupak je postupak liječenja Niemann–Pickove bolesti tip A.
HRP20171165TT 2007-06-06 2017-07-31 Genska terapija za lizosomske bolesti nakupljanja HRP20171165T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US92494707P 2007-06-06 2007-06-06
PCT/US2008/065481 WO2008154198A1 (en) 2007-06-06 2008-06-02 Gene therapy for lysosomal storage diseases
EP08756596.6A EP2158322B1 (en) 2007-06-06 2008-06-02 Gene therapy for lysosomal storage diseases

Publications (1)

Publication Number Publication Date
HRP20171165T1 true HRP20171165T1 (hr) 2017-12-15

Family

ID=40130114

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20171165TT HRP20171165T1 (hr) 2007-06-06 2017-07-31 Genska terapija za lizosomske bolesti nakupljanja

Country Status (14)

Country Link
US (2) US8796236B2 (hr)
EP (2) EP3252161B1 (hr)
AR (1) AR070000A1 (hr)
CY (1) CY1119295T1 (hr)
DK (1) DK2158322T3 (hr)
ES (2) ES2635726T3 (hr)
HR (1) HRP20171165T1 (hr)
HU (1) HUE035779T2 (hr)
IL (3) IL202522A (hr)
LT (1) LT2158322T (hr)
PL (2) PL3252161T3 (hr)
PT (2) PT2158322T (hr)
SI (1) SI2158322T1 (hr)
WO (1) WO2008154198A1 (hr)

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Also Published As

Publication number Publication date
EP2158322A1 (en) 2010-03-03
US20100173979A1 (en) 2010-07-08
EP2158322A4 (en) 2011-06-01
SI2158322T1 (sl) 2017-10-30
US11369693B2 (en) 2022-06-28
IL252898A0 (en) 2017-08-31
CY1119295T1 (el) 2018-02-14
PT2158322T (pt) 2017-08-09
IL252898B (en) 2019-06-30
EP3252161B1 (en) 2021-11-17
ES2635726T3 (es) 2017-10-04
PL3252161T3 (pl) 2022-03-07
IL266734B (en) 2021-10-31
EP3252161A1 (en) 2017-12-06
US8796236B2 (en) 2014-08-05
LT2158322T (lt) 2017-08-25
IL202522A0 (en) 2011-08-01
PL2158322T3 (pl) 2017-10-31
IL202522A (en) 2017-10-31
PT3252161T (pt) 2022-02-01
HUE035779T2 (en) 2018-05-28
WO2008154198A1 (en) 2008-12-18
US20150151007A1 (en) 2015-06-04
AR070000A1 (es) 2010-03-10
DK2158322T3 (en) 2017-08-28
IL266734A (en) 2019-07-31
ES2905616T3 (es) 2022-04-11
EP2158322B1 (en) 2017-05-03

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