DK2499249T3 - Antisense-molekyler og fremgangsmåder til behandling af patologier - Google Patents

Antisense-molekyler og fremgangsmåder til behandling af patologier Download PDF

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DK2499249T3
DK2499249T3 DK10829367.1T DK10829367T DK2499249T3 DK 2499249 T3 DK2499249 T3 DK 2499249T3 DK 10829367 T DK10829367 T DK 10829367T DK 2499249 T3 DK2499249 T3 DK 2499249T3
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exon
antisense oligonucleotide
antisense
skipping
pharmaceutically acceptable
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Stephen Wilton
Sue Fletcher
Abbie Adams
Penny Meloni
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Univ Western Australia
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Claims (20)

1. Antisense-oligonukleotid, der inducerer overspringning af exon 45 i et humant dystrophin-præ-mRNA, hvor antisense-oligonukleotidet er valgt fra gruppen, der består af: i) et antisense-oligonukleotid af 22 baser, der omfatter CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf; ii) et antisense-oligonukleotid af 22 baser, der omfatter CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf; iii) et antisense-oligonukleotid af 25 baser, der omfatter GCC CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf; iv) et antisense-oligonukleotid af 25 baser, der omfatter GCC CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf; v) et antisense-oligonukleotid af 28 baser, der omfatter GCU GCC CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf; vi) et antisense-oligonukleotid af 28 baser, der omfatter GCU GCC CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf; vii) et antisense-oligonukleotid af 31 baser, der omfatter GCC GCU GCC CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf; og viii) et antisense-oligonukleotid af 31 baser, der omfatter GCC GCU GCC CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf.
2. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 22 baser, der omfatter CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf.
3. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 22 baser, der omfatter CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf.
4. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 25 baser, der omfatter GCC CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf.
5. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 25 baser, der omfatter GCC CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf.
6. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 28 baser, der omfatter GCU GCC CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf.
7. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 28 baser, der omfatter GCU GCC CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf.
8. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 31 baser, der omfatter GCC GCU GCC CAA UGC CAU CCU GGA GUU CCU G, eller et farmaceutisk acceptabelt salt deraf.
9. Antisense-oligonukleotid ifølge krav 1, hvor antisense-oligonukleotidet er 31 baser, der omfatter GCC GCU GCC CAA UGC CAU CCU GGA GUU CCU G og omfatter en 5-substitueret pyrimidinbase, eller et farmaceutisk acceptabelt salt deraf.
10. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 9, der omfatter en 5-methylcytosinbase.
11. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 10, hvor oligonukleotidet ikke aktiverer RNase H.
12. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 9, hvor internukleosidbindingerne i rygraden er erstattet med ikke-naturlige internukleosidbindinger.
13. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 9, hvor internukleosidbindingerne er modificerede phosphater.
14. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge krav 13, hvor det modificerede phosphat er valgt blandt methylphosphonater, methylphosphorothioater, phosphoromorpholidater, phosphoropiperazidater og phosphoroamidater.
15. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 9, hvor oligonukleotidet er en peptidnukleinsyre.
16. Antisense-oligonukleotid eller farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 9, hvor oligonukleotidet er kemisk bundet til en eller flere dele eller konjugater, der øger aktiviteten, den cellulære fordeling eller den cellulære optagelse af antisense-oligonukleotidet.
17. Sammensætning, der omfatter et antisense-oligonukleotid eller et farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 16 og et farmaceutisk acceptabelt bæremateriale.
18. Antisense-oligonukleotid eller et farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 16 eller sammensætning ifølge krav 17 til anvendelse i en fremgangsmåde til behandling af muskeldystrofi.
19. Anvendelse af et antisense-oligonukleotid eller et farmaceutisk acceptabelt salt deraf ifølge et hvilket som helst af kravene 1 til 16 eller sammensætning ifølge krav 17 til fremstilling af et medikament til modulering af muskeldystrofi.
20. Antisense-oligonukleotid eller et farmaceutisk acceptabelt salt deraf eller sammensætning til anvendelse ifølge krav 18 eller anvendelse ifølge krav 19, hvor muskeldystrofien er Duchennes muskeldystrofi.
DK10829367.1T 2009-11-12 2010-11-12 Antisense-molekyler og fremgangsmåder til behandling af patologier DK2499249T3 (da)

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AU2009905549A AU2009905549A0 (en) 2009-11-12 Antisense Molecules and Methods for Treating Pathologies
PCT/AU2010/001520 WO2011057350A1 (en) 2009-11-12 2010-11-12 Antisense molecules and methods for treating pathologies

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US (7) US8637483B2 (da)
EP (2) EP2499249B1 (da)
JP (6) JP5963678B2 (da)
KR (8) KR101958491B1 (da)
CN (2) CN105838714B (da)
AU (5) AU2010317599B2 (da)
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CA (1) CA2780563A1 (da)
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