DK2176283T3 - Fremgangsmåder og sammensætninger til behandling af hjernesygdomme - Google Patents

Fremgangsmåder og sammensætninger til behandling af hjernesygdomme Download PDF

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DK2176283T3
DK2176283T3 DK08781733.4T DK08781733T DK2176283T3 DK 2176283 T3 DK2176283 T3 DK 2176283T3 DK 08781733 T DK08781733 T DK 08781733T DK 2176283 T3 DK2176283 T3 DK 2176283T3
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seq
aav
cell
virus
gene
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DK08781733.4T
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Beverly L Davidson
Yong Hong Chen
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Univ Iowa Res Found
Beverly L Davidson
Yong Hong Chen
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
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    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
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    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5058—Neurological cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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    • 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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

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  • Wood Science & Technology (AREA)
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Claims (15)

1. Modificeret adenoassocieret virus (AAV)-capsidprotein, der omfatter et målretningspeptid, hvor målretningspeptidet er fra 3 til 10 aminosyrer langt; og hvor: (i) det modificerede AAV-capsid, der omfatter målretningspeptidet, målretter et AAV mod vildtype-hjernevaskulært endotel, og målretningspeptidet er TLH (SEQ ID NO: 3) eller QSXY (SEQ ID NO: 4), PYFPSLS (SEQ ID NO: 5), YAPLTPS (SEQ ID NO: 6), PLSPSAY (SEQ ID NO: 7), DSPAHPS (SEQ ID NO: 8), GTPTHPS (SEQ ID NO: 9), PDAPSNH (SEQ ID NO: 10), TEPHWPS (SEQ ID NO: 11), SPPLPPK (SEQ ID NO: 12), SPKPPPG (SEQ ID NO: 13), NWSPWDP (SEQ ID NO: 14), DSPAHPS (SEQ ID NO: 15), GWTLHNK (SEQ ID NO: 16), KIPPTLH (SEQ ID NO: 17), ISQTLHG (SEQ ID NO: 18), QSFYILT (SEQ ID NO: 19) eller TTQSEYG (SEQ ID NO: 20) udtrykt i amino-mod-carboxy-orienteringen eller i carboxy-mod-amino-orrenteringen; eller (ii) det modificerede AAV-capsid, der omfatter målretningspeptidet, målretter et AAV mod sygt hjernevaskulært endotel, hvor målretningspeptidet er LXSS (SEQ ID NO: 21) eller SIXA (SEQ ID NO: 23), MLVSSPA (SEQ ID NO: 24), LPSSLQK (SEQ ID NO: 25), PPLLKSS (SEQ ID NO: 26), PXKLDSS (SEQ ID NO: 27), AWTLASS (SEQ ID NO: 28), WPFYGTP (SEQ ID NO: 29), GTFPFLG (SEQ ID NO: 30), GQVPFMG (SEQ ID NO: 31), ANFSILA (SEQ ID NO: 32), GSIWAPA (SEQ ID NO: 33), SIAASFS (SEQ ID NO: 34) eller GMNAFRA (SEQ ID NO: 41) udtrykt i amino-mod-carboxy-orienteringen eller i carboxy-mod-amino-orienteringen.
2. Capsidprotein ifølge krav 1, hvor målretningspeptidet er 3. 4, 5, 6 eller 7 aminosyrer langt, og/eller AAV er AAV2.
3. Nukleinsyresekvens, der koder for det modificerede capsid ifølge krav 1 eller 2.
4. AAV-virus, der indeholder capsidproteinet ifølge krav 1 eller 2.
5. Virusvektor, der omfatter en nukleinsyre, der koder for capsidproteinet ifølge krav 1 eller 2.
6. Virusvektor ifølge krav 5, hvilken virusvektor yderligere omfatter en nukleinsyresekvens, der koder for en nukleinsyre af interesse; fortrinsvis hvor nukleinsyren af interesse er et terapeutisk middel, fortrinsvis hvor det terapeutiske middel er et enzym eller et RNAi-molekyle, fortrinsvis hvor enzymet er β-glucuronidase eller tripeptidylprotease.
7. Celle, der omfatter virusvektoren ifølge krav 5 eller er transduceret med AAV-viruset ifølge krav 4; fortrinsvis hvor cellen er en mammaliacelle, hvor cellen er en human celle in vitro, eller hvor cellen er en ikke-human celle in vitro eller in vivo, fortrinsvis hvor cellen er en endotelcelle, fortrinsvis hvor cellen er en vaskulær endotelcelle.
8. Virusvektor ifølge krav 5 til anvendelse til fremføring af et middel til centralnervesystemet i et ikke-humant individ, hvor anvendelsen omfatter transduktion af vaskulære endotelceller med virusvektoren, således at de transducerede vaskulære endotelceller udtrykker midlet og fremfører midlet til centralnervesystemet hos individet.
9. Virusvektor ifølge krav 5 til anvendelse til medicinsk behandling eller diagnosticering.
10. Anvendelse af virusvektoren ifølge krav 5 eller cellen ifølge krav 7 til fremstilling af et medikament, der er anvendeligt til behandling af en lysosomal ophobningssygdom hos et pattedyr.
11. Celle ifølge krav 7 til anvendelse til medicinsk behandling eller diagnosticering.
12. Capsidprotein ifølge krav 2, hvor AAV er AAV2, og hvor målretningspeptidet er indsat mellem AAV2-V3- aminosyrepositionerne 587 og 588.
13. Virusvektor ifølge krav 6, hvor det terapeutiske middel er et enzym.
14. Virusvektor ifølge krav 13, hvor enzymet er β-glucuronidase eller tripeptidylprotease.
15. Virusvektor ifølge krav 6, hvor det terapeutiske middel er et RNAi-molekyle.
DK08781733.4T 2007-07-14 2008-07-11 Fremgangsmåder og sammensætninger til behandling af hjernesygdomme DK2176283T3 (da)

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PCT/US2008/069866 WO2009012176A2 (en) 2007-07-14 2008-07-11 Methods and compositions for treating brain diseases

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US (5) US8299215B2 (da)
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EP3121190A1 (en) 2017-01-25
WO2009012176A3 (en) 2009-05-07
US8691948B2 (en) 2014-04-08
EP2176283A4 (en) 2011-06-08
WO2009012176A2 (en) 2009-01-22
US8299215B2 (en) 2012-10-30
US20140161777A1 (en) 2014-06-12
US20130142764A1 (en) 2013-06-06
CA3002933A1 (en) 2009-01-22
CA2693712C (en) 2018-05-29
EP2176283B1 (en) 2016-11-02
US20150374781A1 (en) 2015-12-31
US20090162332A1 (en) 2009-06-25
ES2615180T3 (es) 2017-06-05
CA2693712A1 (en) 2009-01-22
US20170348386A1 (en) 2017-12-07
EP2176283A2 (en) 2010-04-21

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