DK2216407T3 - Terapeutiske sammensætninger - Google Patents

Terapeutiske sammensætninger Download PDF

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DK2216407T3
DK2216407T3 DK10005567.2T DK10005567T DK2216407T3 DK 2216407 T3 DK2216407 T3 DK 2216407T3 DK 10005567 T DK10005567 T DK 10005567T DK 2216407 T3 DK2216407 T3 DK 2216407T3
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irna agent
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sequence
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Kallanthottathil Rajeev
Muthiah Manoharan
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Alnylam Pharmaceuticals Inc
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Claims (2)

  1. - 1 - 1. iRNA-middel, der omfatter en sense-streng og en antisense-streng, hvor antisense-strengen og/eller sense- strengen omfatter to eller flere asymmetriske modifikationer, hvilke modifikationer er udvalgt fra følgende gruppe: (a) L-sukre; (b) LNA'er; og (c) konjugatgrupper; hvor modifikationerne forekommer 1-6 nukleotider fra den ene eller den anden ende af strengen, og hvor en asymmetrisk modifikation er en modifikation, der findes på den ene streng men ikke på den anden streng. 2. iRNA-middel ifølge krav 1, hvor mindst én af modifikationerne er en L-sukkermodifikation, og hvor L-sukkermodifikationen er en L-ribose- eller L- arabinosemodifikation. 3. iRNA-middel ifølge krav 2, hvor L-ribose- og L- arabinosemodifikationerne yderligere omfatter en 2'-H, en 2'-OH eller en 2'-0Me. 4. iRNA-middel ifølge krav 2, hvor mindst én af modifikationerne forekommer 1-3 nukleotider fra 3'-enden af antisense-strengen eller sense-strengen eller 1-3 nukleotider fra den ene eller den anden ende af sense-strengen. 5. iRNA-middel ifølge krav 1, hvor iRNA-midlet er et dobbeltstrenget iRNA-middel, der omfatter en dupleksdel med en længde på 15-30 nukleotidpar. 6. iRNA-middel ifølge krav 5, hvor iRNA-midlet omfatter mindst ét 3'-udhæng med en længde på 1-5 nukleotider. 7. iRNA-middel, der omfatter en sense-streng og en antisense-streng, hvor antisense-strengen eller sense-strengen omfatter en 2'-0-alkyl og mindst én L-sukkermodifikation. - 2 - 8. iRNA-middel ifølge krav 7, hvor modifikationerne forekommer 1-6 nukleotider fra den ene eller den anden ende af antisense-strengen eller sense-strengen. 9. iRNA-middel ifølge krav 7, hvor L-sukkermodifikationen er en L-ribose- eller L-arabinosemodifikation. 10. iRNA-middel ifølge krav 9, hvor L-ribose- og L-arabinosemodifikationerne omfatter en 2'-H, en 2'-OH eller en 2'-OMe. 11. iRNA-middel ifølge krav 9, hvor modifikationerne forekommer i 3'-enden af antisense-strengen og/eller sense-strengen . 12. iRNA-middel ifølge krav 8, hvor iRNA-midlet omfatter mindst én 2'-OMe-modifikation og mindst én L-sukkermodifikation. 13. iRNA-middel ifølge krav 8, hvor iRNA-midlet omfatter mindst én 2'-OMe-modifikation, mindst én L-sukkermodifikation og mindst én 2'-5'-koblingsmodifikation. 14. iRNA-middel ifølge krav 1, hvor iRNA-midlet omfatter mindst 2 A:U-grupper i de første 4 terminale par i 5'-enden af antisense-strengen. 15. iRNA-middel ifølge et hvilket som helst af kravene 1 til 14, hvor sense- og antisense-sekvenserne er på forskellige RNA-strenge. 16. iRNA-middel ifølge et hvilket som helst af kravene 1 til 14, hvor sense- og antisense-sekvenserne er på den samme RNA-streng. 17. iRNA-middel ifølge et hvilket som helst af kravene 1 til 16, hvor sense- og antisense-sekvenserne er fuldstændig - 3 - komplementære til hinanden. 18. iRNA-middel ifølge et hvilket som helst af kravene 1 til 17, der yderligere omfatter en kolesteroldel. 19. iRNA-middel ifølge krav 18, hvor kolesteroldelen er koblet til sense-strengen. 20. iRNA-middel ifølge krav 18 eller 19, der yderligere omfatter en anden kolesteroldel. 21. iRNA-middel ifølge krav 20, hvor den anden kolesteroldel er koblet til sense-strengen. 22. iRNA-middel ifølge et hvilket som helst af kravene 1 til 21, hvor iRNA-midlet er mindst 21 nukleotider langt, og dupleksregionen af iRNA'et er 17 til 23 nukleotidpar lang. 23. iRNA-middel ifølge et hvilket som helst af kravene 1 til 21, der har en dupleksregion med en længde på 17 til 23 nukleotidpar og et eller to 3'-udhæng med en længde på 2 subunits.
  2. 24. Farmaceutisk præparat, der omfatter iRNA-midlet ifølge et hvilket som helst af kravene 1 til 23.
DK10005567.2T 2003-03-07 2004-03-08 Terapeutiske sammensætninger DK2216407T3 (da)

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Application Number Priority Date Filing Date Title
US45268203P 2003-03-07 2003-03-07
US45426503P 2003-03-12 2003-03-12
US45496203P 2003-03-13 2003-03-13
US45505003P 2003-03-13 2003-03-13
US46289403P 2003-04-14 2003-04-14
US46377203P 2003-04-17 2003-04-17
US46566503P 2003-04-25 2003-04-25
US46580203P 2003-04-25 2003-04-25
US46961203P 2003-05-09 2003-05-09
US49398603P 2003-08-08 2003-08-08
US49459703P 2003-08-11 2003-08-11
US50634103P 2003-09-26 2003-09-26
US51024603P 2003-10-09 2003-10-09
US51031803P 2003-10-10 2003-10-10
US51845303P 2003-11-07 2003-11-07
EP04718537A EP1605978B1 (en) 2003-03-07 2004-03-08 Therapeutic compositions

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EP (4) EP2239329A1 (da)
AT (1) ATE479752T1 (da)
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