HRP20200647T1 - Genski vektor - Google Patents

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Publication number
HRP20200647T1
HRP20200647T1 HRP20200647TT HRP20200647T HRP20200647T1 HR P20200647 T1 HRP20200647 T1 HR P20200647T1 HR P20200647T T HRP20200647T T HR P20200647TT HR P20200647 T HRP20200647 T HR P20200647T HR P20200647 T1 HRP20200647 T1 HR P20200647T1
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Croatia
Prior art keywords
cell
vector
gene
gene vector
use according
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HRP20200647TT
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English (en)
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
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Ospedale San Raffaele S.R.L.
Fondazione Telethon
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Application filed by Ospedale San Raffaele S.R.L., Fondazione Telethon filed Critical Ospedale San Raffaele S.R.L.
Publication of HRP20200647T1 publication Critical patent/HRP20200647T1/hr

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    • CCHEMISTRY; METALLURGY
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • 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
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • 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
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    • 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
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/555Interferons [IFN]
    • C07K14/56IFN-alpha
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
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    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus

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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)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Claims (14)

1. Genski vektor za uporabu u genskoj terapiji koji sadrži najmanje jednu miRNA ciljanu sekvencu koja odgovara mir-130a ili mir-126 operativno povezanoj s transgenom koji kodira interferon-alfa, lizosomalni enzim galaktocerebrozidaze ili gp91 phox, naznačen time što se genski vektor koristi za sprječavanje ili smanjenje ekspresije transgena u krvotvornoj matičnoj stanici ili krvotvornoj progenitornoj stanici.
2. Genski vektor za uporabu prema patentnom zahtjevu 1 naznačen time što sadrži najmanje jednu miRNA ciljanu sekvencu koja odgovara mir-130a i najmanje jednu miRNA ciljanu sekvencu koja odgovara mir-126.
3. Genski vektor za uporabu prema patentnom zahtjevu 2, naznačen time što je broj kopija miRNA ciljane sekvence koja odgovara mir-130a dvostruki broj kopija miRNA ciljane sekvence koja odgovara mir-126.
4. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što je vektor virusni vektor.
5. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što se vektor može dobiti iz lentivirusa.
6. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što vektor sadrži promotor specifičan za tkivo.
7. Genski vektor za uporabu prema patentnom zahtjevu 6, naznačen time što promotor specifičan za tkivo je CD11b, c-Fes, CYBB ili TEK promotor.
8. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što transgen kodira interferon-alfa.
9. Farmaceutski pripravak za uporabu u genskoj terapiji koji sadrži genski vektor kako je definiran u bilo kojem od patentnih zahtjeva 1-8, naznačen time što se farmaceutski pripravak koristi za sprječavanje ili smanjenje ekspresije transgena krvotvorne matične stanice ili krvotvorne progenitorne stanice.
10. Izolirana stanica za uporabu u genskoj terapiji, naznačena time što je stanica zaražena ili transducirana s genskim vektorom kako je definiran u bilo kojem od patentnih zahtjeva 1-8, te pri čemu je stanica krvotvorna matična stanica ili progenitorna krvotvorna stanica.
11. Genski vektor kako je definiran u bilo kojem od patentnih zahtjeva 1-8, farmaceutski pripravak definiran u patentnom zahtjevu 9 ili stanica kako je definirana u patentnom zahtjevu 10 naznačeni time što su za uporabu za liječenje bolesti odabrane od leukodistrofije globoidnih stanica, kronične granulomatozne bolesti, ozbiljne kombinirane imunodeficijencije (SCID) i solidnih tumora.
12. Genski vektor kako je definiran u bilo kojem od patentnih zahtjeva 1-8, farmaceutski pripravak definiran u patentnom zahtjevu 9 ili stanica kako je definirana u patentnom zahtjevu 10 naznačeni time što su za use povećanje šansi za preživljavanje krvotvornih matičnih stanica ili krvotvornih progenitornih stanica vezanih za gensku terapiju.
13. Uporaba genskog vektora kako je definiran u bilo kojem od patentnih zahtjeva 1-8 ili farmaceutski pripravak definiran u patentnom zahtjevu 9 naznačena time što je za proizvodnju lijeka za gensku terapiju, pri čemu se genski vektor koristi za sprječavanje ili smanjenje ekspresije transgena u krvotvornoj matičnoj stanici ili krvotvornoj progenitornoj stanici.
14. Uporaba izolirane stanice kako je definirana u patentnom zahtjevu 10 naznačena time što je za proizvodnju lijeka za gensku terapiju.
HRP20200647TT 2009-04-30 2020-04-22 Genski vektor HRP20200647T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
EP10723305.8A EP2424571B1 (en) 2009-04-30 2010-04-30 Gene vector
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (1)

Publication Number Publication Date
HRP20200647T1 true HRP20200647T1 (hr) 2020-10-02

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ID=42790671

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HRP20200647TT HRP20200647T1 (hr) 2009-04-30 2020-04-22 Genski vektor

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US (4) US10287579B2 (hr)
EP (2) EP2424571B1 (hr)
JP (2) JP6144911B2 (hr)
KR (1) KR101793615B1 (hr)
CN (1) CN102596255B (hr)
AU (1) AU2010243276B2 (hr)
BR (1) BRPI1010873B1 (hr)
CA (1) CA2759438C (hr)
CY (1) CY1123218T1 (hr)
DK (1) DK2424571T3 (hr)
EA (1) EA023938B1 (hr)
ES (1) ES2786039T3 (hr)
HR (1) HRP20200647T1 (hr)
HU (1) HUE049845T2 (hr)
IL (1) IL215804A (hr)
LT (1) LT2424571T (hr)
MX (1) MX2011011508A (hr)
PL (1) PL2424571T3 (hr)
PT (1) PT2424571T (hr)
SG (2) SG10201610827RA (hr)
SI (1) SI2424571T1 (hr)
WO (1) WO2010125471A2 (hr)

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US20120128643A1 (en) 2012-05-24
IL215804A0 (en) 2012-01-31
US20220333103A1 (en) 2022-10-20
KR20120038403A (ko) 2012-04-23
JP2016028582A (ja) 2016-03-03
US11407996B2 (en) 2022-08-09
LT2424571T (lt) 2020-08-10
BRPI1010873B1 (pt) 2020-04-14
JP2012525141A (ja) 2012-10-22
SG175839A1 (en) 2011-12-29
CA2759438C (en) 2021-03-30
EA201171335A1 (ru) 2012-05-30
AU2010243276A1 (en) 2011-11-17
CA2759438A1 (en) 2010-11-04
US9951328B2 (en) 2018-04-24
JP6144911B2 (ja) 2017-06-07
HUE049845T2 (hu) 2020-10-28
EA023938B1 (ru) 2016-07-29
IL215804A (en) 2016-10-31
AU2010243276B2 (en) 2016-09-15
CY1123218T1 (el) 2021-10-29
SI2424571T1 (sl) 2020-10-30
US20190367911A1 (en) 2019-12-05
KR101793615B1 (ko) 2017-11-03
JP6370278B2 (ja) 2018-08-08
ES2786039T3 (es) 2020-10-08
EP2424571B1 (en) 2020-04-08
DK2424571T3 (da) 2020-05-04
CN102596255A (zh) 2012-07-18
US10287579B2 (en) 2019-05-14
WO2010125471A3 (en) 2010-12-29
WO2010125471A2 (en) 2010-11-04
SG10201610827RA (en) 2017-02-27
PL2424571T3 (pl) 2020-10-19
EP3733214A1 (en) 2020-11-04
PT2424571T (pt) 2020-05-06
BRPI1010873A2 (pt) 2016-03-15
CN102596255B (zh) 2017-10-13
US20180044670A1 (en) 2018-02-15
EP2424571A2 (en) 2012-03-07
MX2011011508A (es) 2012-02-13

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