DK2872625T3 - Fremgangsmåder og sammensætninger til behandling af lysosomale lagringssygdomme - Google Patents

Fremgangsmåder og sammensætninger til behandling af lysosomale lagringssygdomme Download PDF

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DK2872625T3
DK2872625T3 DK13715059.5T DK13715059T DK2872625T3 DK 2872625 T3 DK2872625 T3 DK 2872625T3 DK 13715059 T DK13715059 T DK 13715059T DK 2872625 T3 DK2872625 T3 DK 2872625T3
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cell
artificial sequence
protein
gene
transgene
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Edward J Rebar
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Sangamo Biosciences Inc
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    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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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
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    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

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Claims (13)

1. In vitro fremgangsmåde til generering af en celle, der producerer et protein, der mangler hos et individ med en lysosomal ophobningssygdom, hvilken fremgangsmåde omfatter: integrering af et transgen, der koder for proteinet, i et endogent albuminlocus af cellen ved anvendelse afen ikke-naturligt forekommende nuklease, således at cellen producerer det protein, der mangler i den lysosomale ophobningssygdom.
2. In vitro fremgangsmåden ifølge krav 1, hvor den lysosomale ophobningssygdom er udvalgt fra gruppen bestående af Gauchers, Fabrys, Hunters, Hurlers og Niemann-Picks sygdom.
3. In vitro fremgangsmåden ifølge krav 1 eller krav 2, hvor transgenet koder for et protein, der er udvalgt fra gruppen bestående af glucocerebrosidase, a-galactosidase, iduronat-2-sulfatase, alpha-L-iduronidase og sphingomyelin-phosphodiesterase.
4. In vitro fremgangsmåden ifølge et hvilket som helst af kravene 1 til 3, hvor ekspression af trangenet er drevet afen endogen promoter.
5. In vitro fremgangsmåden ifølge et hvilket som helst af kravene 1 til 4, hvor transgenet er et fusionsprotein, der omfatter aminosyrer, der er kodet for af transgenet, og det endogene locus, hvori transgenet er integreret.
6. In vitro fremgangsmåden ifølge et hvilket som helst af kravene 1 til 5, hvor transgenet koder for et fusionsprotein, der omfatter: (a) et extracellulært domæne af et membranprotein, således, efter ekspression, at fusionsproteinet befinder sig på cellens overflade; og/eller (b) en ligand for en receptor, således, efter ekspression, at fusionsproteinet krydser blod-hjerne-barrieren.
7. In vitro fremgangsmåden ifølge et hvilket som helst af kravene 1 til 6, hvor cellen er udvalgt fra gruppen bestående af et rødt blodlegeme, en levercelle, en muskelcelle og en stamcelle.
8. In vitro fremgangsmåden ifølge krav 7, hvor stamcellen er en hæmopoietisk stamcelle eller induceret pluripotent stamcelle.
9. In vitro fremgangsmåden ifølge et hvilket som helst af kravene 1 til 8, hvor nukleasen er en zinkfingernuklease (ZFN), en TALE-nuklease (TALEN) og/eller et CRISPR/Cas-system.
10. Celle, der er genereret ved hjælp af fremgangsmåden ifølge et hvilket som helst af de foregående krav 1 til 9, til anvendelse i en fremgangsmåde til behandling afen lysosomal ophobningssygdom, hvilken fremgangsmåde omfatter administrering af cellen til et individ med behov derfor.
11. Nuklease eller flere nukleaser og transgen eller flere transgener til anvendelse i en fremgangsmåde til behandling af en lysosomal ophobningssygdom, hvilken fremgangsmåde omfatter administration af den ene eller flere nukleaser og det ene eller flere transgener til et individ med behov derfor til at generere en celle, der producerer et protein, der mangler hos individet, hvor transgenet, der koder for proteinet, integreres i et endogent albuminlocus af cellen ved anvendelse af en ikke-naturligt forekommende nuklease, således at cellen producerer det protein, der mangler i den lysosomale ophobningssygdom.
12. Celle til anvendelse ifølge krav 10, hvor cellen er en isoleret stam- eller præcursorcelle, og fremgangsmåden endvidere omfatter ekspansion og differentiering af stam- eller præcursorcellen forud for administration.
13. Fremgangsmåde ifølge et hvilket som helst af kravene 1 til 9, celle til anvendelse ifølge krav 10 eller 12, eller nukleasen eller flere nukleaser og transgen eller transgener til anvendelse ifølge krav 11, hvor transgenet leveres til cellen ved anvendelse af en viral vektor, såsom en AAV-vektor.
DK13715059.5T 2012-07-11 2013-03-15 Fremgangsmåder og sammensætninger til behandling af lysosomale lagringssygdomme DK2872625T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261670463P 2012-07-11 2012-07-11
US201261704072P 2012-09-21 2012-09-21
PCT/US2013/032381 WO2014011237A1 (en) 2012-07-11 2013-03-15 Methods and compositions for the treatment of lysosomal storage diseases

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DK2872625T3 true DK2872625T3 (da) 2017-02-06

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DK18198271.1T DK3444342T3 (da) 2012-07-11 2013-03-15 Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
DK13715059.5T DK2872625T3 (da) 2012-07-11 2013-03-15 Fremgangsmåder og sammensætninger til behandling af lysosomale lagringssygdomme
DK16198961.1T DK3196301T3 (da) 2012-07-11 2013-03-15 Fremgangsmåder og sammensætninger til behandling af monogene sygdomme

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US (6) US9877988B2 (da)
EP (4) EP2872625B1 (da)
JP (5) JP6225182B2 (da)
AU (1) AU2013289206B2 (da)
CA (1) CA2875618C (da)
DK (3) DK3444342T3 (da)
ES (3) ES2697912T3 (da)
HU (3) HUE051612T2 (da)
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