HRP20150566T1 - Kompozicija za inhibiciju ekspresije gena i njegova upotreba - Google Patents
Kompozicija za inhibiciju ekspresije gena i njegova upotreba Download PDFInfo
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- HRP20150566T1 HRP20150566T1 HRP20150566TT HRP20150566T HRP20150566T1 HR P20150566 T1 HRP20150566 T1 HR P20150566T1 HR P20150566T T HRP20150566T T HR P20150566TT HR P20150566 T HRP20150566 T HR P20150566T HR P20150566 T1 HRP20150566 T1 HR P20150566T1
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- 230000014509 gene expression Effects 0.000 title claims 3
- 230000002401 inhibitory effect Effects 0.000 title claims 2
- 108091034117 Oligonucleotide Proteins 0.000 claims 30
- 150000001875 compounds Chemical class 0.000 claims 18
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 9
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 9
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- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 1
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- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 claims 1
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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Claims (17)
1. Sintetski spoj na bazi oligonukleotida koji sadrži dva oligonukleotida koji su komplementarni jednolančanoj RNA sekvenci, naznačen time da su oligonukleotidi povezani na svojim 5'-krajevima, tako da spoj na bazi oligonukleotida ima dva dostupna 3'-kraja i da spoj na bazi oligonukleotida specifično hibridizira na i inhibira ekspresiju jednolančane RNA sekvence.
2. Spoj na bazi oligonukleotida prema zahtjevu 1, naznačen time da su oligonukleotidi neovisno 15 do 40 nukleotida.
3. Spoj na bazi oligonukleotida prema zahtjevu 1, naznačen time da su oligonukleotidi su povezani jedni s drugima preko nukleotidne veze ili preko ne-nukleotidnog linkera.
4. Spoj na bazi oligonukleotida prema zahtjevu 1, naznačen time da oligonukleotidi sadrže jedan ili više ribonukleotida, deoksiribonukleotida, zaključanih nukleinskih kiselina, arabino šećerne nukleotide ili njihovu kombinaciju.
5. Spoj na bazi oligonukleotida prema zahtjevu 1, naznačen time da je najmanje jedan od oligonukleotida modificiran.
6. Spoj na bazi oligonukleotida prema zahtjevu 5, naznačen time da modificirani oligonukleotid ima najmanje jednu internuklotidnu vezu izabranu iz grupe koja se sastoji od alkilfosfonata, fosforotioata, fosforoditioata, metilfosfonata, i ne-nukleotidnog linkera.
7. Spoj na bazi oligonukleotida prema zahtjevu 5, naznačen time da modificirani oligonukleotid sadrži barem jedan 2'-O-supstituiran nukleotid izabran iz grupe koja se sastoji od 2'-O-metil, 2'-O-metoksi, 2'-O-etoksi, 2'-O-metoksietil, 2'-O-alkil, 2'-O-aril, i 2'-O-alil.
8. Kompozicija koja sadrži spoj na bazi oligonukleotida prema bilo kojem od zahtjeva 1 do 7 i fiziološki prihvatljiv nosač.
9. Spoj na bazi oligonukleotida prema bilo kojem od zahtjeva 1 do 7 za upotrebu za inhibiciju ekspresije gena.
10. Spoj na bazi oligonukleotida prema zahtjevu 9, naznačen time da je oligonukleotid komplementaran RNA koja kodira Bcl-2, EGFR, mdm2, MyD88, PCSK9, survivin, VEGF, ili TLR.
11. Spoj na bazi oligonukleotida prema zahtjevu 10, naznačen time da je TLR izabran između TLR2, TLR3, TLR4, TLR5, TLR6, TLR7, TLR8 i TLR9.
12. Spoj na bazi oligonukleotida prema zahtjevu 9, naznačen time da je svaki od oligonukleotida komplementaran različitoj regiji jednoloančane RNA sekvence.
13. Spoj na bazi oligonukleotida prema bilo kojem od zahtjeva 1 do 7 za upotrebu u postupku za terapijski tretman sisavca koji ima bolest ili poremećaj posredovan proteinom, naznačen time da su oligonukleotidi komplementarni jednolančanoj RNA koja kodira protein.
14. Spoj na bazi oligonukleotida prema bilo kojem od zahtjeva 1 do 7 za upotrebu u postupku prevencije bolesti ili poremećaja posredovanog proteinom kod sisavca koji ima rizik za razvoj bolesti iki poremećaja, naznačen time da su oligonukleotidi komplementarni jednolančanoj RNA koja kodira protein.
15. Spoj na bazi oligonukleotida prema zahtjevu 13 ili 14, naznačen time da je bolest ili poremećaj izabran između raka, autoimunog poremećaja, upale dišnih putova, upalnih poremećaja, infektivnih bolesti, kožnih poremećaja, alergija, astme, bakterijske, virusne ili gljivične infekcije, ili bolesti uzrokovane patogenom.
16. Spoj na bazi oligonukleotida prema bilo kojem od zahtjev 9 do 15, koji se primjenjuje u kombinaciji s jednim ili više cjepiva, antigena, protutijela, citotoksičnih sredstava, alergena, antibiotika, TLR agonista, TLR antagonista, siRNA molekula, rniRNA molekula, antisense nukleotida, aptamera, proteina, peptida, vektora genske terapije, DNA cjepiva, adjuvansa, antagonista proteinske aktivnosti, kinaznih inhibitora, kemoterapeutika, ciljanih terapijskih sredstava, ko-stimulatornih molekula ili njihovih kombinacija.
17. Kompozicija prema zahtjevu 8 koja nadalje sadrži jedno ili više cjepiva, antigena, protutijela, citotoksičnih sredstava, kemoterapeutskih sredstava, inhibitora kinaze, alergena, antibiotika, agonista, antagonista, antisense oligonukleotida, ribozima, RNA molekula, siRNA molekula, miRNA molekula, aptamera, proteina, vektora genske terapije, DNA cjepiva, adjuvansa, ko-stimulatornih molekula ili njihove kombinacije.
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- 2010-08-26 JP JP2012526977A patent/JP5805088B2/ja not_active Expired - Fee Related
- 2010-08-26 EP EP10749570.7A patent/EP2470656B1/en active Active
- 2010-08-26 US US12/869,185 patent/US20110082186A1/en not_active Abandoned
- 2010-08-26 CA CA2772352A patent/CA2772352A1/en not_active Abandoned
- 2010-08-26 DK DK10749570.7T patent/DK2470656T3/da active
- 2010-08-26 SI SI201030947T patent/SI2470656T1/sl unknown
- 2010-08-26 EP EP17190602.7A patent/EP3272870A1/en not_active Withdrawn
- 2010-08-26 ES ES15165966.1T patent/ES2646097T3/es active Active
- 2010-08-26 PL PL10749570T patent/PL2470656T3/pl unknown
- 2010-08-26 ES ES10749570.7T patent/ES2538347T3/es active Active
- 2010-08-26 WO PCT/US2010/046781 patent/WO2011031520A1/en active Application Filing
- 2010-08-26 CN CN201080048450.8A patent/CN102712926B/zh active Active
- 2010-08-26 CN CN201410573396.9A patent/CN104673795A/zh active Pending
- 2010-08-26 PT PT107495707T patent/PT2470656E/pt unknown
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ES2646097T3 (es) | 2017-12-12 |
CN102712926A (zh) | 2012-10-03 |
US20120016004A1 (en) | 2012-01-19 |
CN104673795A (zh) | 2015-06-03 |
EP2470656A1 (en) | 2012-07-04 |
PT2470656E (pt) | 2015-07-16 |
ES2538347T3 (es) | 2015-06-19 |
US8431544B1 (en) | 2013-04-30 |
JP5805088B2 (ja) | 2015-11-04 |
CA2772352A1 (en) | 2011-03-17 |
EP2960333B1 (en) | 2017-10-04 |
DK2470656T3 (da) | 2015-06-22 |
PL2470656T3 (pl) | 2015-08-31 |
HK1210223A1 (en) | 2016-04-15 |
CN102712926B (zh) | 2015-04-29 |
SI2470656T1 (sl) | 2015-07-31 |
SMT201500140B (it) | 2015-09-07 |
WO2011031520A1 (en) | 2011-03-17 |
US20110082186A1 (en) | 2011-04-07 |
EP2960333A1 (en) | 2015-12-30 |
EP3272870A1 (en) | 2018-01-24 |
HUE026020T2 (en) | 2016-05-30 |
JP2013507905A (ja) | 2013-03-07 |
EP2470656B1 (en) | 2015-05-06 |
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