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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DK10829367.1T
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Stephen Wilton
Sue Fletcher
Abbie Adams
Penny Meloni
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Univ Western Australia
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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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/11—Antisense
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    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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) KR102113306B1 (da)
CN (2) CN105838714B (da)
AU (5) AU2010317599B2 (da)
BR (2) BR122023023194A2 (da)
CA (1) CA2780563A1 (da)
CY (1) CY1121198T1 (da)
DK (1) DK2499249T3 (da)
ES (1) ES2693459T3 (da)
HR (1) HRP20181824T1 (da)
HU (1) HUE040445T2 (da)
IL (5) IL297299A (da)
LT (1) LT2499249T (da)
NZ (2) NZ716534A (da)
PL (1) PL2499249T3 (da)
PT (1) PT2499249T (da)
RS (1) RS58079B1 (da)
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