DK2561073T3 - Aav-vektorer målrettet mod centralnervesystemet og fremgangsmåder til anvendelse deraf - Google Patents

Aav-vektorer målrettet mod centralnervesystemet og fremgangsmåder til anvendelse deraf Download PDF

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DK2561073T3
DK2561073T3 DK11772784.2T DK11772784T DK2561073T3 DK 2561073 T3 DK2561073 T3 DK 2561073T3 DK 11772784 T DK11772784 T DK 11772784T DK 2561073 T3 DK2561073 T3 DK 2561073T3
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Guangping Gao
Hongyan Wang
Zuoshang Xu
Hongwei Zhang
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Univ Massachusetts
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Claims (11)

1.
rAAV, der omfatter (i) et capsidprotein, der omfatter sekvensen ifølge SEQ ID NO: 9, og (ii) en nukleinsyre, der omfatter en promotor, der er operabelt koblet med et transgen, til anvendelse til behandling af amyotrofisk lateral sklerose (ALS); hvor rAVV'en er til fremføring til vævet i centralnervesystemet (CNS) hos en person ved intratekal administration og eventuelt også fremføring ved intracerebral administration.
2.
rAVV til anvendelse ifølge krav 1 hvor rAVV'en er til fremføring til CNS-vævet hos en person ved intratekal administration og ved intracerebral administration.
3.
rAVV til anvendelse ifølge krav 2, hvor den intracerebrale administration er intraventrikulær administration.
4.
rAVV til anvendelse ifølge et hvilket som helst af kravene 1 til 3, hvor den intratekale administration er i cervikalregionen af rygmarvskanalen hos personen.
5.
rAVV til anvendelse ifølge et hvilket som helst af kravene 1 til 3, hvor den intratekale administration er i torakalregionen af rygmarvskanalen hos personen.
6. rAVV til anvendelse ifølge et hvilket som helst af kravene 1 til 3, hvor den intratekale administration er i lumbalregionen af rygmarvskanalen hos personen. 7. rAVV til anvendelse ifølge et hvilket som helst af kravene 1 til 6, hvor dosen af rAVV til intratekal eller intracerebral administration er i et interval fra 1010 genomkopier til 1011 genomkopier. 8. rAVV til anvendelse ifølge et hvilket som helst af kravene 1 til 7, hvor transgenet koder for et inhibitor-RNA, der binder specifikt til SODl-mRNA og inhiberer ekspression af SODI hos personen. 9. rAVV til anvendelse ifølge krav 8, hvor inhibitor-RNA'et har en sekvens ifølge SEQ ID NO: 26. 10. rAVV til anvendelse ifølge et hvilket som helst af kravene 1 til 9, hvor rAVV'en er formuleret i en sammensætning, der omfatter et farmaceutisk acceptabelt bæremateriale.
DK11772784.2T 2010-04-23 2011-04-22 Aav-vektorer målrettet mod centralnervesystemet og fremgangsmåder til anvendelse deraf DK2561073T3 (da)

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US32762710P 2010-04-23 2010-04-23
PCT/US2011/033616 WO2011133890A1 (en) 2010-04-23 2011-04-22 Cns targeting aav vectors and methods of use thereof

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DK2561073T3 true DK2561073T3 (da) 2016-12-12

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US (8) US9102949B2 (da)
EP (6) EP2561073B1 (da)
JP (6) JP5963743B2 (da)
CA (3) CA3066596A1 (da)
DK (2) DK2561073T3 (da)
ES (2) ES2698203T3 (da)
WO (1) WO2011133890A1 (da)

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