HRP20160261T1 - Komplementarni antagonisti i njihova uporaba - Google Patents

Komplementarni antagonisti i njihova uporaba Download PDF

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HRP20160261T1
HRP20160261T1 HRP20160261TT HRP20160261T HRP20160261T1 HR P20160261 T1 HRP20160261 T1 HR P20160261T1 HR P20160261T T HRP20160261T T HR P20160261TT HR P20160261 T HRP20160261 T HR P20160261T HR P20160261 T1 HRP20160261 T1 HR P20160261T1
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oligomer
oligomer according
nucleotides
modified
sugar part
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HRP20160261TT
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Frank Baas
Kees Fluiter
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Regenesance B.V.
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Claims (14)

1. Jednolančani antisense oligomer duljine od 10 do 50 nukleotida koji ima graničnu nukleobaznu sekvenciju s bar 80% sekvencijske identičnosti s komplementarnom regijom nukleinske kiseline koja kodira KOMPLEMENTARNU KOMPONENTU 6 (C6) sekvence predstavljene SEQ ID NO: 1; oligomer sadrži najmanje jedan analog nukleotida a koji ima sposobnost reduciranja razine C6 mRNA ekspresije u sisavaca za najmanje 20% kako je određeno pomoću qPCR testa, naznačen time da je oligomer targetiran na nukleotide 112-152, 433-473, 546 -586, 706-746 ili 1015-1055 iz ATG start mjesta SEQ ID NO: 1.
2. Oligomer prema zahtjevu 1, naznačen time da oligomer nadalje sadrži barem jedno od modificirane internukleotide spojnice i modificirane nukleobaze.
3. Oligomer prema zahtjevu 1, naznačen time da je analog nukleotida modificirani šećerni dio odabran iz skupine koja se sastoji od: 2'-O-metoksietil modificirani šećerni dio, 2'-metoksi modificirani šećerni dio, 2'-O-alkil modificirani šećera dio i biciklički šećerni dio.
4. Oligomer prema zahtjevu 3, naznačen time da je biciklički šećerni dio monomer zaključane nukleinske kiseline (LNA).
5. Oligomer prema zahtjevu 2, naznačen time da je modificirana internukleotidna spojnica fosforotioatna internukleotidna spojnica.
6. Oligomer prema zahtjevu 2, naznačen time da je modificirana nukleobaza 5-metilcitozin.
7. Oligomer prema zahtjevu 1, naznačen time da je oligomer između 12 do 45 nukleotida duljine.
8. Oligomer prema zahtjevu 1, naznačen time da je oligomer između 10 do 18 nukleotida duljine.
9. Oligomer prema zahtjevu 1, naznačen time da je oligomer targetiran na nukleotide 132, 453, 566, 726 ili 1035 iz ATG start mjesta SEQ ID NO: 1.
10. Oligomer prema zahtjevu 1 ili 9, naznačen time da oligomer ima najmanje 90%, 96%, 97%, 98% ili 99% sekvencijske identičnosti s KOMPLEMENTARNOM KOMPONENTOM C6 sekvence predstavljene SEQ ID NO: 1.
11. Farmaceutski pripravak koji sadrži oligomer prema zahtjevu 1 ili 2 i farmaceutski prihvatljivo otapalo, sol ili pomoćno sredstvo.
12. Uporaba najmanje jednog oligomera prema zahtjevu 1 za proizvodnju lijeka za liječenje stanja koje zahtijeva regeneraciju aksona.
13. Uporaba najmanje jednog od oligomera prema zahtjevu 1 za proizvodnju lijeka za liječenje kroničnog demijelinizirajućeg stanja.
14. Uporaba prema zahtjevu 13, naznačena time da je kronično demijelinizirajuće stanje multipla skleroza, kronična demijelinacijska neuropatija, HMSN (CMT) tipa bolesti IA i IB, Alzheimerova bolest, Huntingtonova bolest, Charcot-Marie-Tooth bolest, amiotrofna lateralna skleroza (ALS), Guillain-Barre sindrom (GBS, također poznat kao akutna upalna demijelinacijska polineuropatija ili AIDP), leukodistrofija, ili Parkinsonova bolest.
HRP20160261TT 2008-07-10 2016-03-14 Komplementarni antagonisti i njihova uporaba HRP20160261T1 (hr)

Applications Claiming Priority (3)

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US7950108P 2008-07-10 2008-07-10
PCT/NL2009/050418 WO2010005310A2 (en) 2008-07-10 2009-07-10 Complement antagonists and uses thereof
EP09788222.9A EP2320925B1 (en) 2008-07-10 2009-07-10 Complement antagonists and uses thereof

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HRP20160261T1 true HRP20160261T1 (hr) 2016-06-03

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US (2) US8703730B2 (hr)
EP (1) EP2320925B1 (hr)
JP (1) JP2011527568A (hr)
KR (1) KR20110044983A (hr)
CN (2) CN102149390A (hr)
AU (1) AU2009270020B2 (hr)
CA (1) CA2730203C (hr)
CY (1) CY1117476T1 (hr)
DK (1) DK2320925T3 (hr)
ES (1) ES2566008T3 (hr)
HR (1) HRP20160261T1 (hr)
HU (1) HUE027169T2 (hr)
IL (1) IL210506A (hr)
NZ (1) NZ591057A (hr)
PL (1) PL2320925T3 (hr)
SI (1) SI2320925T1 (hr)
SM (1) SMT201600098B (hr)
WO (1) WO2010005310A2 (hr)

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JP2011527568A (ja) 2011-11-04
IL210506A (en) 2015-06-30
NZ591057A (en) 2012-11-30
US8703730B2 (en) 2014-04-22
AU2009270020B2 (en) 2014-09-25
PL2320925T3 (pl) 2016-06-30
AU2009270020A1 (en) 2010-01-14
ES2566008T3 (es) 2016-04-08
CN103740713A (zh) 2014-04-23
US9089555B2 (en) 2015-07-28
CY1117476T1 (el) 2017-04-26
AU2009270020A2 (en) 2011-01-27
CA2730203A1 (en) 2010-01-14
WO2010005310A3 (en) 2010-04-15
HUE027169T2 (en) 2016-10-28
KR20110044983A (ko) 2011-05-03
SMT201600098B (it) 2016-04-29
CN102149390A (zh) 2011-08-10
DK2320925T3 (en) 2016-03-21
WO2010005310A2 (en) 2010-01-14
US20150211003A9 (en) 2015-07-30
US20110171176A1 (en) 2011-07-14
CA2730203C (en) 2016-12-13
SI2320925T1 (sl) 2016-05-31
EP2320925B1 (en) 2015-12-23
US20140301982A1 (en) 2014-10-09
EP2320925A2 (en) 2011-05-18

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