DK2529020T3 - Skalerbar fremstillingsplatform til oprensning af viralvektorer og oprensede virale vektorer til anvendelse i genterapi - Google Patents

Skalerbar fremstillingsplatform til oprensning af viralvektorer og oprensede virale vektorer til anvendelse i genterapi Download PDF

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DK2529020T3
DK2529020T3 DK11737508.9T DK11737508T DK2529020T3 DK 2529020 T3 DK2529020 T3 DK 2529020T3 DK 11737508 T DK11737508 T DK 11737508T DK 2529020 T3 DK2529020 T3 DK 2529020T3
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aav
vector
particles
factor
vectors
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John Fraser Wright
Guang Qu
Bernd Hauck
Katherine High
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Childrens Hospital Philadelphia
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Claims (11)

1. Fremgangsmåde til oprensning af bona fide adeno-associerede viruspartikler (AAV), der omfatter et transgen, der koder for et terapeutisk protein eller fragment deraf fra et AAV-præparat omfattende AAV-vektorpartikler, tomme kapsider og værtscelleurenheder, hvilken fremgangsmåde omfatter, at: a) høste celler og cellesupernatant omfattende AAV partikler; b) koncentrere cellerne og supernatanten via tværfiltrering (ENG: tangential flow filtration); c) lysere cellerne ved mikrofluidisering for at danne et lysat; d) filtrere for derved at opnå klaring af lysatet fra trin c); e) oprense AAV-partikler omfattende bona fide AAV-vektorpartikler ved ionbytnings-søjlekromatografi til fremstilling af et eluat og eventuelt koncentrere eluatet ved tværfiltrering (ENG: tangential flow filtration); f) blande eluatet med cæsiumchlorid og underkaste blandingen gradient ultracentri-fugering for at adskille bona fide AAV-vektorer fra AAV-vektorrelaterede urenheder; g) opsamle AAV-partikler omfattende bona-fide AAV-vektorpartikler adskilt i trin f) og underkaste en bufferudveksling ved tværfiltrering (ENG: tangential flow filtration); h) formulere nævnte AAV-partikler omfattende bona-fide AAV-vektorpartikler fra trin g) med overfladeaktivt middel for at opnå en AAV-partikelformulering; i) filtrere formuleringen fra trin h) for at fjerne resterende urenheder, hvor bona fide AAV-vektorpartiklerne er til stede i det resulterende filtrat i en mængde på mindst 90%.
2. Fremgangsmåde ifølge krav 1, hvor bona fide AAV-vektorpartiklerne er afledt fra en AAV valgt fra gruppen bestående af AAV1, AAV2, AAV5, AAV6, AAV8 og AAV9.
3. Fremgangsmåde ifølge krav 1, hvor transgenet koder for en nukleinsyre valgt fra gruppen bestående af et siRNA, et antisensmolekyle og et miRNA, et ribozym og et shRNA.
4. Fremgangsmåde ifølge krav 1, hvor transgenet koder for et genprodukt valgt fra gruppen bestående af insulin, glucagon, væksthormon (GH), parathyroidhormon (PTH), væksthormonfrigivende faktor (GRF), follikelstimulerende hormon (FSH), luteiniserende hormon (LH), menneskelig choriongonadotropin (hCG), vaskulær endotel vækstfaktor (VEGF), angiopoietiner, angiostatin, granulocytkolonistimulerende faktor (GCSF), erythropoietin (EPO), bindevævsvækstfaktor (CTGF), basisfibroblastvækstfaktor (bFGF), sur fibroblastvækstfaktor (aFGF), epidermal vækstfaktor (EGF), transformerende vækstfaktor alpha (TGFa), blodpladeafledt vækstfaktor (PDGF), insulinvækstfaktorer I og II (IGF-I og IGF-II), TGFp, activiner, inhibiner, knoglemorfogenprotein (BMP), nervevækstfaktor (NGF), hjerneafledt neurotrofisk faktor (BDNF), neurotrophiner NT-3 og NT4/5, ciliær neurotrofisk faktor (CNTF), glialcellelinieafledt neurotrofisk faktor (GDNF), neurturin, agrin, netrin-1 og netrin-2, hepatocytvækstfaktor (HGF), ephriner, noggin, sonic hedgehog og tyrosinhydro-xylase.
5. Fremgangsmåde ifølge krav 1, hvor transgenet koder for et genprodukt valgt fra gruppen bestående af thrombopoietin (TPO), interleukiner (IL1 til IL-17), monocytkemoat-traktant protein, leukæmiinhiberende faktor, granulocyt-makrofagkolonistimulerende faktor, Fas-ligand tumor nekrosefaktorer α og β, interferoner a, β og y stamcellefaktor, flk-2/flt3 ligand, IgG, IgM, IgA, IgD og IgE, kimære immunglobuliner, humaniserede antistoffer, enkeltkædeantistoffer, T-cellereceptorer, kimære T-cellereceptorer, enkeltkæde-T-cellereceptorer, klasse I og klasse II MHC-molekyler.
6. Fremgangsmåde ifølge krav 1, hvor transgenet omfatter en nukleinsyre kodende for et protein, der er egnet til korrektion af medfødte fejl af metabolisme valgt fra gruppen bestående af carbamoylsyntetase I, ornithintranscarbamylase, arginosuccinatsyntetase, arginosuccinat-lyase, arginase, fumarylacetacetathydrolase, phenylalaninhydroxylase, al-pha-1 antitrypsin, glucose-6-phosphatase, porphobilinogen deaminase, faktor V, faktor VIII, faktor IX, cystathion beta-syntase, forgrenet ketoacid decarboxylase, albumin, isova-leryl-CoA dehydrogenase, propionyl-CoA carboxylase, methylmalonyl-CoA mutase, gluta-ryl-CoA dehydrogenase, insulin, beta-glycosidase, pyruvatcarboxylat, hepatisk phosphorylase, phosphorylase-kinase, glycin-decarboxylase, RPE65, H-protein, T-protein, en cystisk fibrose transmembranregulator (CFTR)-sekvens og en dystrophin-cDNA sekvens.
7. Fremgangsmåde ifølge krav 6, hvor genproduktet er faktor VIII.
8. Fremgangsmåde ifølge krav 1, hvor tomme kapsider er til stede i filtratet fra trin i) i en mængde på 10% eller mindre.
9. Fremgangsmåde ifølge krav 1, hvor bona fide AAV-vektorpartikler er til stede i filtratet fra trin i) i en mængde på mindst 95%.
10. Fremgangsmåde ifølge krav 9, hvor tomme kapsider er til stede i filtratet fra trin i) i en mængde på 5% eller mindre.
11. Fremgangsmåde ifølge krav 6, hvor genproduktet er faktor IX.
DK11737508.9T 2010-01-28 2011-01-25 Skalerbar fremstillingsplatform til oprensning af viralvektorer og oprensede virale vektorer til anvendelse i genterapi DK2529020T3 (da)

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