DK1771206T3 - Lipider, lipid-komplekser og anvendelse deraf - Google Patents

Lipider, lipid-komplekser og anvendelse deraf Download PDF

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DK1771206T3
DK1771206T3 DK05742844.3T DK05742844T DK1771206T3 DK 1771206 T3 DK1771206 T3 DK 1771206T3 DK 05742844 T DK05742844 T DK 05742844T DK 1771206 T3 DK1771206 T3 DK 1771206T3
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lipid
cell
group
compound
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DK05742844.3T
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Jörg Kaufmann
Oliver Keil
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Silence Therapeutics Gmbh
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    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/06—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
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    • C07C209/50—Preparation of compounds containing amino groups bound to a carbon skeleton by reduction of carboxylic acids or esters thereof in presence of ammonia or amines, or by reduction of nitriles, carboxylic acid amides, imines or imino-ethers by reduction of carboxylic acid amides
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Claims (53)

1. Forbindelse ifølge formel (I),
(i) hvor Ri og R2 er hver og uafhængigt valgt fra gruppen omfattende alkyl; hvor termen alkyl henviser til et mættet alifatisk radikal eller et mono- eller polyumættet alifatisk carbonhydrid-radikal; n er 1, 2, 3, eller 4; R3 er et acyl valgt fra gruppen omfattende lysyl, ornithyl, 2,4-diaminobutyryl, histidyl og en acyl-del ifølge formel (II),
(Π) hvor m er et hvilket som helst heltal fra 1 til 3 og Y" er en farmaceutisk acceptabel anion.
2. Forbindelsen ifølge krav 1, hvor Ri og R2 er hver og uafhængigt valgt fra gruppen omfattende lauryl, myristyl, palmityl og oleyl.
3. Forbindelsen ifølge et hvilket som helst af kravene 1 og 2, hvor Ri er lauryl og R2 er myristyl; eller Ri er palmityl og R2 er oleyl.
4. Forbindelsen ifølge et hvilket som helst af kravene 1 til 3, hvor m er 1 eller 2.
5. Forbindelsen ifølge et hvilket som helst af kravene 1 til 4, hvor forbindelsen er et kationisk lipid, fortrinsvis i forbindelse med en anion Y\
6. Forbindelsen ifølge et hvilket som helst af kravene 1 til 5, hvor Y- er valgt fra gruppen omfattende halogenider, acetat og trifluoracetat.
7. Forbindelsen ifølge et hvilket som helst af kravene 1 til 6, hvor forbindelsen er valgt fra gruppen omfattende - · -arginyl-2,3-diamino propionsyre-N-palmityl-N-oleyl-amidtrihydrochlorid
- · -arginyl-2,3-diamino propionsyre-N-lauryl-N-myristyl-amidtrihydrochlorid
og - •-arginyl-lysin-N-lauryl-N-myristyl-amidtrihydrochlorid
8. Sammensætning omfattende som en lipid-komponent en forbindelse ifølge et hvilket som helst af kravene 1 til 7, og en bærer.
9. Sammensætningen ifølge krav 8, hvor sammensætningen omfatter en yderligere konstituent.
10. Farmaceutisk sammensætning omfattende en forbindelse ifølge et hvilket som helst af kravene 1 til 7 og en farmaceutisk aktiv forbindelse og fortrinsvis en farmaceutisk acceptabel bærer.
11. Sammensætningen ifølge et hvilket som helst af kravene 8 til 10, hvor den farmaceutisk aktive forbindelse og/eller den yderligere konstituent er valgt fra gruppen omfattende peptider, proteiner, oligonukleotider, polynukleotider og nukleinsyrer.
12. Sammensætningen ifølge krav 11, hvor proteinet er et antistof, fortrinsvis et monoklonalt antistof.
3. Sammensætningen ifølge krav 11, hvor nukleinsyren er valgt fra gruppen omfattende DNA, RNA, PNA og LNA.
14. Sammensætningen ifølge et hvilket som helst af kravene 11 eller 13, hvor nukleinsyren er en funktionel nukleinsyre, hvor fortrinsvis den funktionelle nukleinsyre er valgt fra gruppen omfattende RNAi, siRNA, siNA, antisense-nukleinsyre, ribozymer, aptamerer og spiegelmerer.
15. Sammensætningen ifølge et hvilket som helst af kravene 8 til 14, yderligere omfattende mindst en hjælper-lipid-komponent, hvor fortrinsvis hjælper-lipid-komponenten er valgt fra gruppen omfattende phospholipider og steroider.
16. Sammensætningen ifølge krav 15, hvor hjælper-lipid-komponenten er valgt fra gruppen omfattende 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamin og 1,2-dioleyl-sn-glycero-3-phosphoethanolamin.
17. Sammensætningen ifølge et hvilket som helst af kravene 15 til 16, hvor indholdet af hjælper-lipid-komponenten er fra 20 mol% til 80 mol% af sammensætningens samlede lipidindhold.
18. Sammensætningen ifølge krav 17, hvor indholdet af hjælper-lipid-komponenten er fra 35 mol% til 65 mol%.
19. Sammensætningen ifølge et hvilket som helst af kravene 16 til 18, hvor lipidet er ·-arginyl-2,3-diamino propionsyre-N-palmityl-N-oleyl-amidtrihydrochlorid, og hjælper-lipidet er 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamin.
20. Sammensætningen ifølge krav 19, hvor lipidet er 50 mol% og hjælper-lipidet er 50 mol% af sammensætningens samlede lipidindhold.
21. Sammensætningen ifølge et hvilket som helst af kravene 8 til 20, hvor sammensætningen indeholder mindst to hjælper-lipider.
22. Sammensætningen ifølge krav 21, hvor mindst et hjælper-lipid omfatter en del, der er valgt fra gruppen omfattende en PEG-del, en HEG-del, en polyhydroxyethylstivelses (polyHES)-del og en polypropylen-del, hvorved sådan del fortrinsvis tilvejebringer en molekylevægt fra 500 til 10000 Da, mere fortrinsvis fra 2000 til 5000 Da.
23. Sammensætningen ifølge kravene 21 eller 22, hvor hjælper-lipidet omfattende PEG-delen er valgt fra gruppen omfattende 1,2-distearoyl-sn-glycero-3-phosphoethanolamin, 1,2-dialkyl-sn-glycero-3-phosphoethanolamin; og Ceram id-PEG.
24. Sammensætningen ifølge krav 23, hvor PEG-delen har en molekylevægt fra 500 Da til 10000 Da, fortrinsvis fra 2000 til 5000 Da, mere fortrinsvis en molekylevægt på 2000 Da.
25. Sammensætningen ifølge krav 24, hvor sammensætningen omfatter som lipid-komponenten · -arginyl-2,3-diamino propionsyre-N-palmityl-N-oleyl-amidtrihydrochlorid, som et første hjælper-lipid 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamin og som et andet hjælper-lipid 1,2-disteroyl-sn-glycero-3-phosphoethanolamin-PEG2000.
26. Sammensætningen ifølge krav 25, hvor indholdet af det andet hjælper-lipid er fra 0,05 mol% til 4,9 mol%, fortrinsvis 1 til 3 mol%.
27. Sammensætningen ifølge krav 26, hvor indholdet af lipidet er fra 45 mol% til 50 mol%, indholdet af det første hjælper-lipid er fra 45 til 50 mol% og, med det forbehold at der er et PEGyleret andet hjælper-lipid, hvor indholdet af det andet hjælper-lipid er fra 0,1 mol% til 5 mol%, fortrinsvis fra 1 til 4 mol% og mere fortrinsvis 2 %, hvor summen af indholdet af lipidet, af lipidet, af det første hjælper-lipid og af det andet hjælper-lipid er 100 mol% og hvor summen af det første hjælper-lipid og det andet hjælper-lipid er 50 mol%.
28. Sammensætningen ifølge et hvilket som helst af kravene 21 til 27 indeholdende: a) 50 mol% af ·-arginyl-2,3-diamino propionsyre-N-palmityl-N-oleyl-amidtrihydrochlorid, 48 mol% af 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamin; og 2 mol% 1,2-distearoyl-sn-glycero-3-phosphoethanolamin-PEG2000. eller b) 50 mol% af ·-L-arginyl-2,3-L-diamino propionsyre-N-palmityl-N-oleyl-amidtrihydroclorid, 49 mol% 1,2-diphytanoyl-sn-glycero-3-phosphoethanolamin; og 1 mol% N(Carbonyl-methoxypolyethylenglycol-2000)-1,2-distearoyl-sn-glycero-3-phosphoethanolamin, fortrinsvis natriumsaltet deraf.
29. Sammensætningen ifølge et hvilket som helst af krav 8 til 28, hvor den funktionelle nukleinsyre er en dobbeltstrenget ribonukleinsyre, hvor sammensætningen yderligere omfatter en nukleinsyre, fortrinsvis en funktionel nukleinsyre, der er mere fortrinsvis en dobbeltstrenget ribonukleinsyre og mest fortrinsvis en nukleinsyre valgt fra gruppen omfattende RNAi, siRNA, siNA, antisense-nukleinsyre og ribozym, hvor fortrinsvis molforholdet af RNAi til kationisk lipid er fra 0 til 0,075, fortrinsvis fra 0,02 til 0,05 og endnu mere fortrinsvis 0,037.
30. Sammensætningen ifølge et hvilket som helst af kravene 8 til 29, hvor forbindelsen og/eller hjælper-lipid-komponenten er til stede som en dispersion i et vandigt medium.
31. Sammensætningen ifølge et hvilket som helst af kravene 8 til 29, hvor forbindelsen og/eller hjælper-lipid-komponenten er til stede som en opløsning i et vandblandbart solvent, hvor fortrinsvis solventet er valgt fra gruppen omfattende ethanol og tert.-butanol.
32. Sammensætningen ifølge et hvilket som helst af kravene 8 til 31, hvor den funktionelle nukleinsyre er en dobbeltstrenget ribonukleinsyre, fortrinsvis en nukleinsyre valgt fra gruppen omfattende RNAi, siRNA, siNA, antisense-nukleinsyre og ribozym, og hvor fortrinsvis molforholdet af RNAi til kationisk lipid er fra 0 til 0,075, fortrinsvis fra 0,02 til 0,05 og endnu mere fortrinsvis 0,037.
33. Sammensætningen ifølge et hvilket som helst af kravene 8 til 32, fortrinsvis 29 til 32, hvor sammensætningen indeholder en nukleinsyre, hvor ladningsforholdet af nukleinsyre-backbone-phosphater til kationiske lipid-nitrogen-atomer er fra 1:1,5-7, fortrinsvis 1:4.
34. Sammensætningen ifølge et hvilket som helst af kravene 8 til 33, fortrinsvis 29 til 33, hvor størrelsen af partiklerne i dispersionen er 120 nm.
35. Sammensætningen ifølge et hvilket som helst af kravene 8 til 34, fortrinsvis 29 til 34, hvor dispersionen er en stam-dispersion indeholdende 1 til 100 μΜ siRNA, hvor fortrinsvis stam-dispersionen fortyndes in vivo eller in vitro med 1: 100 til 1:10000, mere fortrinsvis 1:1000.
36. Anvendelse af en forbindelse ifølge et hvilket som helst af kravene 1 til 7 eller en sammensætning ifølge et hvilket som helst af kravene 8 til 35, til fremstilingen af et medikament.
37. Anvendelse ifølge krav 36, hvor medikamentet er til behandlingen af kræft og/eller kardiovaskulær-relaterede sygdomme.
38. Anvendelse ifølge krav 37, hvor medikamentet er til behandlingen af kræft, hvor fortrinsvis kræften er valgt fra gruppen omfattende faste og ikke-faste tumorer og hvor mere fortrinsvis den faste tumor er valgt fra gruppen omfattende kræft i bugspytkirtlen, brystkræft, prostatakræft, lungekræft, kolonkræft og hepatocellulær karcinom.
39. Anvendelse ifølge krav 37 eller 38, hvor kræften involverer en proces valgt fra gruppen omfattende angiogenese og neoangiogenese.
40. Anvendelse ifølge krav 36 eller 37, hvor medikamentet er til administrering af nukleinsyren til en celle valgt fra gruppen omfattende endotelceller, epitelceller og tumorceller, hvor fortrinsvis cellen er en endotelcelle.
41. Anvendelse ifølge krav 40, hvor endotelcellerne er endotelceller af vaskulatur.
42. Anvendelse ifølge krav 41, hvor vaskulaturen er vaskulatur stammende fra neoangiogenese, fortrinsvis tumor-associeret neoangiogenese, eventuelt hvor vaskulaturen er valgt fra gruppen omfattende lever-vaskulatur, hjerte-vaskulatur, nyre-vaskulatur, bugspytkirtel-vaskulatur og lunge-vaskulatur.
43. Anvendelse ifølge et hvilket som helst af kravene 36 til 42, hvor medikamentet er til systemisk administration.
44. Anvendelse ifølge et hvilket som helst af krav 36 til 42, hvor medikamentet er til lokal administration.
45. Anvendelse ifølge et hvilket som helst af kravene 36 til 44, hvor medikamentet er et diagnostisk middel.
46. Anvendelse af en forbindelse ifølge et hvilket som helst af kravene 1 til 7 eller en sammensætning ifølge et hvilket som helst af kravene 8 til 35, til anvendelse in vitro som et overførselsmiddel.
47. Anvendelse ifølge krav 46, hvor overførselsmidlet overfører en farmaceutisk aktiv komponent og/eller en yderligere konstituent ind i en celle, fortrinsvis en pattedyrscelle og mere fortrinsvis en human celle.
48. Anvendelse ifølge krav 47, hvor cellen er en endotelcelle, fortrinsvis en vaskulær-associeret endotelcelle.
49. In w'fro-fremgangsmåde til overførsel af en farmaceutisk aktiv forbindelse og/eller en yderligere konstituent ind i en celle eller over en membran, fortrinsvis en cellemembran, omfattende de følgende trin: - tilvejebringelse af cellen eller membranen; - tilvejebringelse af en forbindelse ifølge et hvilket som helst af kravene 1 til 7; - tilvejebringelse af den farmaceutisk aktive forbindelse og/eller den yderligere konstituent; og - at bringe cellen eller membranen i kontakt med den farmaceutisk aktive forbindelse og/eller den yderligere konstituent, og forbindelsen ifølge et hvilket som helst af kravene 1 til 7.
50. In w'fro-fremgangsmåde til overførsel af en farmaceutisk aktiv forbindelse og/eller en yderligere konstituent ind i en celle eller over en membran, fortrinsvis en cellemembran, der tilvejebringer de følgende trin: - tilvejebringelse af cellen eller membranen; - tilvejebringelse af en sammensætning ifølge et hvilket som helst af kravene 8 til 35; og - at bringe cellen eller membranen i kontakt med sammensætningen ifølge et hvilket som helst af kravene 8 til 35.
51. Fremgangsmåden ifølge kravene 49 og 50, hvor den farmaceutisk aktive forbindelse omfatter som et yderligere trin: - at detektere den farmaceutisk aktive forbindelse og/eller den yderligere konstituent i cellen og/eller ud over membranen.
52. Forbindelse ifølge et hvilket som helst af kravene 1 til 7 eller en sammensætning ifølge et hvilket som helst af kravene 8 til 35 til anvendelse som et medikament i behandlingen af en sygdom.
53. Forbindelsen eller sammensætning ifølge krav 52, hvor medikamentet er egnet til systemisk administration, fortrinsvis systemisk administration til et hvirveldyr.
54. Forbindelsen eller sammensætning ifølge krav 53, hvor hvirveldyret er et pattedyr, mere fortrinsvis et pattedyr valgt fra gruppen omfattende mus, rotte, marsvin, kat, hund, abe og menneske.
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