DK2581448T3 - Tricyclo-phosphorothioat-DNA - Google Patents
Tricyclo-phosphorothioat-DNA Download PDFInfo
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- DK2581448T3 DK2581448T3 DK11185129T DK11185129T DK2581448T3 DK 2581448 T3 DK2581448 T3 DK 2581448T3 DK 11185129 T DK11185129 T DK 11185129T DK 11185129 T DK11185129 T DK 11185129T DK 2581448 T3 DK2581448 T3 DK 2581448T3
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- dna
- nucleic acid
- acid molecule
- mrna
- exon
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Claims (13)
1. Nukleinsyremolekyle omfattende tricyclo-nukleosider forbundet af internukleosid phosphorothioat forbindelser (3'-0PS-0-5' forbindelser).
2. Nukleinsyremolekyle ifølge krav 1, omfattende mellem 3 og 50 nukleotider.
3. Nukleinsyremolekyle ifølge krav 1 eller 2, hvilket er komplementært til en målsekvens.
4. Nukleinsyremolekyle ifølge krav 3, hvor nukleinsyremolekylet er et antisense oligonukleotid komplementært til en del af et RNA som kodet for af et gen.
5. Nukleinsyremolekyle ifølge krav 1, omfattende eller bestående af sekvensen 5'-AACCTCGGCTTACCT-3' (SEQ ID NR.:1).
6. Fremgangsmåde til syntetisering af et tricyclo-phosphorothioat-DNA-molekyle, fremgangsmåden omfattende: a) at tilvejebringe et første tricyclo-nukleosid bundet til en faststoffasestøtte, hvilket første nukleotid har en beskyttet 5'-OH-gruppe; b) at afbeskytte 5'-gruppen for at danne en fri 5'-OH-gruppe; c) at omsætte den frie 5'-OH-gruppe med en 5'-beskyttet tricyclonukleosid-S'-O-cyanoethyl-^N-diisopropylaminophosphoramidit monomer for at danne en internukleosid phosphoramidit forbindelse mellem det første og et andet tricyclo-nukleosid; og d) at svovlbehandle internukleosid-phosphoramidit-gruppen for at danne en phosphorothioat internukleosid-forbindelse mellem det første og andet tricyclo-nukleosid.
7. Farmaceutisk sammensætning omfattende et nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 4, i en farmaceutisk acceptabel bærer.
8. Nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 5, til anvendelse som et lægemiddel.
9. Nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 4, til anvendelse i behandlingen af en hjertesygdom.
10. Nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 5, til anvendelse i behandlingen afen neuromuskulær eller muskuloskeletal sygdom.
11. Nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 5, til anvendelse i behandlingen af en centralnervesystem-sygdom.
12. Nukleinsyremolekyle til anvendelse ifølge et hvilket som helst af kravene 9 til 11, hvor hjerte-, CNS-, neuromuskulær eller muskuloskeletal sygdom resulterer fra en ændring af et gen, hvor ændringen er en i-ramme mutation af et exon, en mutation som forstyrrer den translatoriske læseramme af genet, og tc-DNA'et gør det lettere at springe et exon over for at gendanne læserammen; en skadelig mutation som kan kompenseres ved inklusionen af et atypisk exon i mRNA'et som koder for genet, og tc-DNA'et er komplementært til en ISS eller TSL til stede i et præ-mRNA som er kodet for af genet og gør inklusion lettere af et atypisk exon, eller en mutation som resulterer i tilstedeværelsen af skadelig 3’ CUG amplifikation(er) i et mRNA som kodet for af genet.
13. Nukleinsyremolekyle til anvendelse ifølge krav 10, til behandlingen af Duchenne muskeldystrofi, spinal muskelatrofi, eller Steinerts myotonisk dystrofi.
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| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP11185129.1A EP2581448B1 (en) | 2011-10-13 | 2011-10-13 | Tricyclo-phosphorothioate DNA |
Publications (1)
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|---|---|
| DK2581448T3 true DK2581448T3 (da) | 2015-04-27 |
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| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK11185129T DK2581448T3 (da) | 2011-10-13 | 2011-10-13 | Tricyclo-phosphorothioat-DNA |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US9738891B2 (da) |
| EP (2) | EP2581448B1 (da) |
| JP (1) | JP6181653B2 (da) |
| CN (1) | CN104245935B (da) |
| AU (1) | AU2012322903B2 (da) |
| BR (1) | BR112014009066B8 (da) |
| CA (2) | CA2776651C (da) |
| DK (1) | DK2581448T3 (da) |
| ES (2) | ES2535654T3 (da) |
| IL (1) | IL231983B (da) |
| IN (1) | IN2014DN03463A (da) |
| PL (1) | PL2581448T3 (da) |
| PT (1) | PT2581448E (da) |
| WO (1) | WO2013053928A1 (da) |
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2011
- 2011-10-13 PL PL11185129T patent/PL2581448T3/pl unknown
- 2011-10-13 PT PT111851291T patent/PT2581448E/pt unknown
- 2011-10-13 DK DK11185129T patent/DK2581448T3/da active
- 2011-10-13 ES ES11185129.1T patent/ES2535654T3/es active Active
- 2011-10-13 EP EP11185129.1A patent/EP2581448B1/en active Active
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2012
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- 2012-10-12 BR BR112014009066A patent/BR112014009066B8/pt active IP Right Grant
- 2012-10-12 CN CN201280057688.6A patent/CN104245935B/zh active Active
- 2012-10-12 CA CA2851970A patent/CA2851970A1/en not_active Abandoned
- 2012-10-12 US US14/351,733 patent/US9738891B2/en active Active
- 2012-10-12 EP EP12770503.6A patent/EP2766479B1/en active Active
- 2012-10-12 AU AU2012322903A patent/AU2012322903B2/en active Active
- 2012-10-12 WO PCT/EP2012/070349 patent/WO2013053928A1/en not_active Ceased
- 2012-10-12 ES ES12770503.6T patent/ES2651216T3/es active Active
- 2012-10-12 JP JP2014535113A patent/JP6181653B2/ja active Active
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Also Published As
| Publication number | Publication date |
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| CA2776651C (en) | 2021-06-08 |
| BR112014009066B8 (pt) | 2021-02-23 |
| CN104245935A (zh) | 2014-12-24 |
| PT2581448E (pt) | 2015-05-21 |
| ES2651216T3 (es) | 2018-01-25 |
| EP2581448B1 (en) | 2015-01-28 |
| EP2581448A1 (en) | 2013-04-17 |
| BR112014009066B1 (pt) | 2020-08-25 |
| BR112014009066A2 (pt) | 2017-04-18 |
| CA2851970A1 (en) | 2013-04-18 |
| JP6181653B2 (ja) | 2017-08-16 |
| ES2535654T3 (es) | 2015-05-13 |
| IN2014DN03463A (da) | 2015-06-05 |
| WO2013053928A1 (en) | 2013-04-18 |
| JP2015501144A (ja) | 2015-01-15 |
| PL2581448T3 (pl) | 2015-08-31 |
| CA2776651A1 (en) | 2013-04-13 |
| IL231983A0 (en) | 2014-05-28 |
| AU2012322903A1 (en) | 2014-05-29 |
| IL231983B (en) | 2018-04-30 |
| HK1186205A1 (en) | 2014-03-07 |
| CN104245935B (zh) | 2018-05-08 |
| EP2766479A1 (en) | 2014-08-20 |
| US9738891B2 (en) | 2017-08-22 |
| BR112014009066A8 (pt) | 2018-01-09 |
| US20140296323A1 (en) | 2014-10-02 |
| AU2012322903B2 (en) | 2017-09-14 |
| EP2766479B1 (en) | 2017-09-13 |
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