DK1620544T3 - MODIFICEREDE iRNA-MIDLER - Google Patents

MODIFICEREDE iRNA-MIDLER Download PDF

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DK1620544T3
DK1620544T3 DK04759946.9T DK04759946T DK1620544T3 DK 1620544 T3 DK1620544 T3 DK 1620544T3 DK 04759946 T DK04759946 T DK 04759946T DK 1620544 T3 DK1620544 T3 DK 1620544T3
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target
strand
sequence
irna agent
irna
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DK04759946.9T
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Muthiah Manoharan
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Alnylam Pharmaceuticals Inc
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Priority claimed from PCT/US2004/007070 external-priority patent/WO2004080406A2/en
Priority claimed from PCT/US2004/010586 external-priority patent/WO2004090108A2/en
Priority claimed from PCT/US2004/011255 external-priority patent/WO2004091515A2/en
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
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Publication of DK1620544T3 publication Critical patent/DK1620544T3/da

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    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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Claims (30)

1. iRNA-middel, der omfatter en første streng og en anden streng, hvor mindst én subunit med formel (I) er inkorporeret i mindst én af strengene:
(I) hvor: X er N(CO)R7 eller NR7; Y er CR9R10; Z er fraværende; én af R1, R2, R3, R4, R9 og R10 er 0Ra, og én af R1, R2, R3, R4, R9 og R10 er (CH2)nORb, eller én af R1, R2, R3, R4, R9 og R10 er (CH2)nORa, og én af R1, R2, R3, R4, R9 og R10 er 0Rb, hvor de resterende af R1, R2, R3, R4, R9 og R10 er H; hver af R5 og R6 uafhængigt er H eller Ci-C6alkyl, der eventuelt er substitueret med 1-3 R13; R7 er Rd; eller Ci-C2oalkyl substitueret med NRcRd eller NHC (0) Rd; R13 er hydroxy, Ci-C4alkoxy eller halogen; Ra er Rb er hver af A og C uafhængigt er 0 eller S; B er OH, 0“ eller
Rc er H eller Ci-C6alkyl;
Rd er en ligand valgt blandt et cholesterolradikal; et carbohydratradikal; og cholesterol; og n er 1-4; hvor subunitten er anbragt i 3'- eller 5'-enden af den første streng eller den anden streng.
2. Forbindelse ifølge krav 1, hvor R1 er (CH2)nORb, og R3 er 0Ra.
3. Forbindelse ifølge krav 2, hvor R1 og R3 er cis.
4. Forbindelse ifølge krav 2, hvor R1 og R3 er trans.
5. Forbindelse ifølge krav 2, hvor n er 1.
6. Forbindelse ifølge krav 2, hvor A er 0.
7. Forbindelse ifølge krav 2, hvor A er S.
8. Forbindelse ifølge krav 1, hvor R1 er (CH2)n0Ra, og R3 er 0Rb.
9. Forbindelse ifølge krav 2, hvor R7 er (CH2)sNHRd eller (CH2) sNHC (0) Rd.
10. Forbindelse ifølge krav 1, hvor R1 er 0Rb, og R3 er (CH2)nORa.
11. Forbindelse ifølge krav 1, hvor R1 er 0Ra, og R3 er (CH2)nORb.
12. Forbindelse ifølge krav 11, hvor R1 og R3 er cis.
13. Forbindelse ifølge krav 11, hvor R1 og R3 er trans.
14. Forbindelse ifølge krav 11, hvor n er 1.
15. Forbindelse ifølge krav 1, hvor R1 er (CH2)nORb, og R9 er 0Ra.
16. Forbindelse ifølge krav 15, hvor R1 og R9 er cis.
17. Forbindelse ifølge krav 15, hvor R1 og R9 er trans.
18. Forbindelse ifølge krav 15, hvor n er 1.
19. Forbindelse ifølge krav 1, hvor R1 er 0Ra, og R9 er (CH2)nORb.
20. Forbindelse ifølge krav 19, hvor R1 og R9 er cis.
21. Forbindelse ifølge krav 19, hvor R1 og R9 er trans.
22. Forbindelse ifølge krav 19, hvor n er 1.
23. Forbindelse ifølge krav 1, hvor R1 er (CH2)nORa, og R9 er ORb.
24. Forbindelse ifølge krav 1, hvor R1 er ORb, og R9 er (CH2)nORa.
25. Forbindelse ifølge krav 1, hvor R3 er (CH2)nORb, og R9 er ORa.
26. Forbindelse ifølge krav 1, hvor R3 er (CH2)nORa, og R9 er ORb.
27. Forbindelse ifølge krav 1, hvor R3 er ORa, og R9 er (CH2)nORb.
28. Forbindelse ifølge krav 1, hvor R3 er ORb, og R9 er (CH2)nORa.
29. Farmaceutisk sammensætning, der omfatter et iRNA-middel ifølge krav 1 og et farmaceutisk acceptabelt bæremateriale.
30. Kit, der omfatter et iRNA-middel ifølge krav 1, en steril beholder, hvori iRNA-midlet er indeholdt, og en brugsanvisning.
DK04759946.9T 2003-04-17 2004-04-16 MODIFICEREDE iRNA-MIDLER DK1620544T3 (da)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
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
US50341403P 2003-09-15 2003-09-15
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
PCT/US2004/007070 WO2004080406A2 (en) 2003-03-07 2004-03-08 Therapeutic compositions
PCT/US2004/010586 WO2004090108A2 (en) 2003-04-03 2004-04-05 Irna conjugates
PCT/US2004/011255 WO2004091515A2 (en) 2003-04-09 2004-04-09 iRNA CONJUGATES
PCT/US2004/011829 WO2004094595A2 (en) 2003-04-17 2004-04-16 MODIFIED iRNA AGENTS

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DK1620544T3 true DK1620544T3 (da) 2019-01-14

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DK04759946.9T DK1620544T3 (da) 2003-04-17 2004-04-16 MODIFICEREDE iRNA-MIDLER

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US (7) US7745608B2 (da)
EP (5) EP1620544B1 (da)
JP (1) JP4597976B2 (da)
AU (2) AU2004233092C9 (da)
CA (1) CA2522637C (da)
DK (1) DK1620544T3 (da)
ES (1) ES2702942T3 (da)
WO (1) WO2004094595A2 (da)

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