HRP20200762T1 - Virioni rekombinantnog adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe - Google Patents

Virioni rekombinantnog adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe Download PDF

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HRP20200762T1
HRP20200762T1 HRP20200762TT HRP20200762T HRP20200762T1 HR P20200762 T1 HRP20200762 T1 HR P20200762T1 HR P20200762T T HRP20200762T T HR P20200762TT HR P20200762 T HRP20200762 T HR P20200762T HR P20200762 T1 HRP20200762 T1 HR P20200762T1
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Croatia
Prior art keywords
seq
capsid protein
cell
virion
raav
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HRP20200762TT
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English (en)
Inventor
David V. Schaffer
Ryan R. Klimczak
James T. Koerber
John G. Flannery
Deniz DALKARA MOUROT
Meike VISEL
Leah C.T. BYRNE
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The Regents Of The University Of California
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Publication of HRP20200762T1 publication Critical patent/HRP20200762T1/hr

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Claims (15)

1. Virion rekombinantnog adeno-povezanog virusa (rAAV) ili farmaceutski pripravak koji sadrži navedeni virion, za upotrebu u postupku liječenja očne bolesti kod pojedinca kojem je to potrebno, pri čemu pripravak sadrži farmaceutski prihvatljiv ekscipijent, te pri čemu virion rekombinantnog adeno-povezanog virusa (rAAV) sadrži: a) varijantu AAV kapsidnog proteina, pri čemu varijanta AAV kapsidnog proteina sadrži umetanje peptida u GH petlju kapsidnog proteina u odnosu na odgovarajući roditeljski AAV kapsidni protein, pri čemu umetanje sadrži aminokiselinsku sekvencu odabranu od LALGETTRPA (SEQ ID NO:45); LANETITRPA (SEQ ID NO:46), LAKAGQANNA (SEQ ID NO:47), LAKDPKTTNA (SEQ ID NO:48), KDTDTTR (SEQ ID NO:57), RAGGSVG (SEQ ID NO:58), AVDTTKF (SEQ ID NO:59), STGKVPN (SEQ ID NO:60), LAKDTDTTRA (SEQ ID NO:61), LARAGGSVGA (SEQ ID NO:62), LAAVDTTKFA (SEQ ID NO:63), i LASTGKVPNA (SEQ ID NO:64); i b) heterolognu nukleinsku kiselinu koja sadrži nukleotidnu sekvencu koja kodira genski proizvod; pri čemu varijanta kapsidnog proteina inficira stanicu mrežnice.
2. Virion rAAV ili farmaceutski pripravak za uporabu u skladu s patentnim zahtjevom 1, naznačen time što je mjesto umetanja unutar aminokiselina 570-611 AAV2 kapsidnog proteina navedenog u SEQ ID NO:1, ili unutar odgovarajućeg položaja u kapsidnom proteinu drugog AAV serotipa.
3. Virion rAAV ili farmaceutski pripravak za uporabu u skladu s patentnim zahtjevom 1 ili 2, naznačen time što je stanica mrežnice fotoreceptor, ganglijska stanica mrežnice, Müllerova stanica, bipolarna stanica, amakrinska stanica, vodoravna stanica, ili stanica pigmentiranog epitela mrežnice.
4. Virion rAAV ili farmaceutski pripravak za uporabu u skladu s bilo kojim od patentnih zahtjeva 1 do 3, naznačen time što je navedeno liječenje s intraokularnom injekcijom.
5. Virion rAAV ili farmaceutski pripravak za uporabu u skladu s bilo kojim od patentnih zahtjeva 1 do 4, naznačen time što je navedeno liječenje s intravitrealnom injekcijom.
6. Virion rAAV ili farmaceutski pripravak za uporabu u skladu s bilo kojim od patentnih zahtjeva 1 do 5, naznačen time što je očna bolest glaukom, retinitis pigmentoza, makularna degeneracija, retinoshiza, Leberova prirođena amauroza, dijabetička retinopatija, akromotopsija, ili sljepoća za boju.
7. Virion rekombinantnog adeno-povezanog virusa (rAAV) koji sadrži: a) varijantu AAV kapsidnog proteina, pri čemu varijanta AAV kapsidnog proteina sadrži umetanje peptida u GH petlju kapsidnog proteina u odnosu na odgovarajući roditeljski AAV kapsidni protein, pri čemu umetanje sadrži aminokiselinsku sekvencu odabranu od LALGETTRPA (SEQ ID NO:45); LANETITRPA (SEQ ID NO:46), LAKAGQANNA (SEQ ID NO:47), LAKDPKTTNA (SEQ ID NO:48), LAKDTDTTRA (SEQ ID NO:61), LARAGGSVGA (SEQ ID NO:62), LAAVDTTKFA (SEQ ID NO:63), i LASTGKVPNA (SEQ ID NO:64), i pri čemu varijanta kapsidnog proteina donosi infektivnost stanici mrežnice; i b) heterolognu nukleinsku kiselinu koja sadrži nukleotidnu sekvencu koja kodira genski proizvod.
8. Virion rAAV prema patentnom zahtjevu 7, naznačen time što je mjesto umetanja unutar aminokiselina 570-611 AAV2 kapsidnog proteina ili unutar odgovarajućeg položaja u kapsidnom proteinu drugog AAV serotipa.
9. Virion rAAV prema bilo kojem od patentnih zahtjeva 7 do 8, naznačen time što je stanica mrežnice fotoreceptor, ganglijska stanica mrežnice, Müllerova stanica, bipolarna stanica, amakrinska stanica, vodoravna stanica, ili stanica pigmentiranog epitela mrežnice.
10. Virion rAAV prema patentnom zahtjevu 7, naznačen time što je mjesto umetanja unutar aminokiselina 587-588 AAV2 kapsidnog proteina navedeno u SEQ ID NO:1 ili unutar odgovarajućeg položaja u kapsidnom proteinu drugog AAV serotipa.
11. Virion rAAV prema bilo kojem od patentnih zahtjeva 7 do 10, naznačen time što je genski produkt interferentna RNK, aptamer, ili polipeptid.
12. Virion rAAV prema patentnom zahtjevu 11, naznačen time što je polipeptid odabran iz skupine koja sadrži: neurotrofni faktor koji potiče iz glija, faktor rasta fibroblasta 2, neurturin, cilijarni neurotrofni faktor, faktor rasta živaca, neurotrofni faktor koji potiče iz mozga, faktor rasta epiderme, rodopsin, X-vezani inhibitor apoptoze, retinoshizin, RPE65, protein-1 koji reagira na retinitis pigmentosa GTPaze, periferin, periferin-2, rodopsin, i Sonic hedgehog.
13. Farmaceutski pripravak, naznačen time što sadrži: a) virion rekombinantnog adeno-povezanog virusa prema bilo kojem od zahtjeva 7 do 12; i b) farmaceutski prihvatljiv ekscipijent.
14. Izolirana nukleinska kiselina naznačena time što sadrži nukleotidnu sekvencu koja kodira varijantu kapsidnog proteina adeno-povezanog virusa (AAV), pri čemu je varijanta AAV kapsidnog proteina definirana kao u bilo kojem od zahtjeva 7 do 12.
15. Izolirana, genetski modificirana stanica domaćina koja sadrži nukleinsku kiselinu prema patentnom zahtjevu 14.
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