DK3118311T3 - Antisense-nukleinsyre - Google Patents

Antisense-nukleinsyre Download PDF

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DK3118311T3
DK3118311T3 DK15761392.8T DK15761392T DK3118311T3 DK 3118311 T3 DK3118311 T3 DK 3118311T3 DK 15761392 T DK15761392 T DK 15761392T DK 3118311 T3 DK3118311 T3 DK 3118311T3
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antisense oligomer
nucleotide sequence
pharmaceutically acceptable
hydrate
exon
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DK15761392.8T
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Tatsushi Wakayama
Haruna Seo
Youhei Satou
Shin'ichi Takeda
Tetsuya Nagata
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Nippon Shinyaku Co Ltd
Nat Center Neurology & Psychiatry
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    • 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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Claims (15)

1. Antisense-oligomer, der er valgt fra en gruppe bestående af (a) til (c) nedenfor, eller et farmaceutisk acceptabelt salt eller hydrat deraf: (a) en antisense-oligomer, der omfatter en nukleotidsekvens ifølge SEQ ID NO: 1 eller 2; (b) en antisense-oligomer, der består af en nukleotidsekvens med deletion, substitution, insertion og/eller addition af 1 til 5 nukleotider i nukleotidsekvensen ifølge SEQ ID NO: 1 eller 2 og har en aktivitet til at bevirke overspringning af den 5E exon i det humane dy strophin-gen; og (c) antisense-oligomeren, der har en nukleotidsekvens med mindst 80 % identitet med en nukleotidsekvens ifølge SEQ ID NO: 1 eller 2 og har en aktivitet til at bevirke overspringning af den 5E exon i det humane dystrophin-gen.
2. Antisense-oligomer, der er udvalgt fra en gruppe bestående af (e) til (g) nedenfor, eller et farmaceutisk acceptabelt salt eller hydrat deraf: (e) en antisense-oligomer, der består af en nukleotidsekvens ifølge SEQ ID NO: 1 eller 2; (f) en antisense-oligomer, der består af en nukleotidsekvens med deletion, substitution, insertion og/eller addition af 1 til 3 nukleotider i nukleotidsekvensen ifølge SEQ ID NO: 1 eller 2 og har en aktivitet til at bevirke overspringning af den 5E exon i det humane dystrophin-gen; og (g) en antisense-oligomer der består af en nukleotidsekvens med mindst 80 % identitet med en nukleotidsekvens ifølge SEQ ID NO: 1 eller 2 og har en aktivitet til at bevirke overspringning af den 51. exon i det humane dystrophin-gen.
3. Antisense-oligomer, der er udvalgt fra en gruppe bestående af (j) nedenfor, eller et farmaceutisk acceptabelt salt eller hydrat deraf: (j) en antisense-oligomer, der har en nukleotidsekvens med mindst 90 % identitet med en nukleotidsekvens ifølge SEQ ID NO: 1 eller 2 og har en aktivitet til at bevirke overspringning af den 5E exon i det humane dystrophin-gen.
4. Antisense-oligomer ifølge et hvilket som helst af kravene 1 til 3, der er et oligonukleotid, eller et farmaceutisk acceptabelt salt eller hydrat deraf.
5. Antisense-oligomer ifølge krav 4, eller et farmaceutisk acceptabelt salt eller hydrat deraf, hvor sukkerdelen og/eller den phosphat-bindende region af mindst ét nukleotid, der udgør oligonukleotidet, er modificeret.
6. Antisense-oligomer ifølge krav 4 eller 5, eller et farmaceutisk acceptabelt salt eller hydrat deraf, hvor sukkerdelen af mindst ét nukleotid, der udgør oligonukleotidet, er en ribose, hvori 2'-OH-gruppen er udskiftet med en hvilken som helst valgt fra gruppen bestående af OR, R, ROR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br og I (hvor R er alkyl eller aryl og R' er alkylen).
7. Antisense-oligomer ifølge et hvilket som helst af krav 4 til 6, eller et farmaceutisk acceptabelt salt eller hydrat deraf, hvor den phosphat-bindende region af mindst ét nukleotid, der udgør oligonukleotidet, er en hvilken som helst valgt fra gruppen bestående af en phosphorthioat-binding, en phosphordithioat-binding, en alkylphosphonat-binding, en phosphoramidat-binding og en borphosphat-binding.
8. Antisense-oligomer ifølge et hvilket som helst af kravene 1 til 3, der er en morpholin-oligomer eller et farmaceutisk acceptabelt salt eller hydrat deraf.
9. Antisense-oligomer ifølge krav 8, der er en phosphordiamidat-morpholin-oligomer eller et farmaceutisk acceptabelt salt eller hydrat deraf.
10. Antisense-oligomer ifølge krav 8 eller 9, eller et farmaceutisk acceptabelt salt eller hydrat deraf, hvor 5'-enden er en hvilken som helst af nedenstående kemiske formler (1) til (3):
11. Farmaceutisk sammensætning til behandling af muskeldystrofi, der som aktiv bestanddel omfatter antisense-oligomeren eller et farmaceutisk acceptabelt salt eller hydrat deraf ifølge et hvilket som helst af kravene 1 til 10.
12. Farmaceutisk sammensætning ifølge krav 11, der omfatter en farmaceutisk acceptabel bærer.
13. Antisense-oligomer, eller et farmaceutisk acceptabelt salt eller hydrat deraf ifølge et hvilket som helst af kravene 1 til 10, til anvendelse i behandling af muskeldystrofi.
14. Antisense-oligomer, eller et farmaceutisk acceptabelt salt eller hydrat deraf til anvendelse ifølge krav 13, hvor patienten med muskeldystrofi i behandlingen er en patient med deletioner af nukleotider i exonerne 29-50, 50, 45-50, 48- 50, 49-50, 52, 52-63, 13-50, 19-50, 43-50 eller 47-50.
15. Antisense-oligomer, eller et farmaceutisk acceptabelt salt eller hydrat deraf til anvendelse ifølge krav 13 eller 14, hvor patienten er et menneske.
DK15761392.8T 2014-03-12 2015-03-11 Antisense-nukleinsyre DK3118311T3 (da)

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JP2014048897 2014-03-12
PCT/JP2015/057180 WO2015137409A1 (ja) 2014-03-12 2015-03-11 アンチセンス核酸

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US (4) US9988629B2 (da)
EP (2) EP3514234A1 (da)
JP (1) JP6606488B2 (da)
KR (1) KR102335801B1 (da)
CN (2) CN110951732A (da)
AU (2) AU2015227733B2 (da)
BR (1) BR112016020618B1 (da)
CA (1) CA2939948A1 (da)
CY (1) CY1121322T1 (da)
DK (1) DK3118311T3 (da)
ES (1) ES2710802T3 (da)
HR (1) HRP20190235T1 (da)
HU (1) HUE042283T2 (da)
IL (1) IL247663B (da)
LT (1) LT3118311T (da)
MX (1) MX2016011538A (da)
MY (1) MY193708A (da)
NZ (1) NZ724836A (da)
PH (1) PH12016501761B1 (da)
PL (1) PL3118311T3 (da)
PT (1) PT3118311T (da)
RS (1) RS58573B1 (da)
RU (2) RU2702424C2 (da)
SG (1) SG11201607095RA (da)
SI (1) SI3118311T1 (da)
TR (1) TR201901939T4 (da)
TW (2) TWI672314B (da)
UA (1) UA120849C2 (da)
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