Cyclohexane lipidoids for nucleic acid transfection and use thereof

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F61388963%3A_____%2F25%3A00618123" target="_blank" >RIV/61388963:_____/25:00618123 - isvavai.cz</a>

Výsledek na webu

<a href="https://worldwide.espacenet.com/patent/search/family/082939774/publication/AU2022314101B2?q=AU2022314101B2" target="_blank" >https://worldwide.espacenet.com/patent/search/family/082939774/publication/AU2022314101B2?q=AU2022314101B2</a>

DOI - Digital Object Identifier

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Jazyk výsledku

angličtina

Název v původním jazyce

Cyclohexane lipidoids for nucleic acid transfection and use thereof

Popis výsledku v původním jazyce

The present invention relates to a lipidoid of general formula I, wherein X is selected from -C(=O)NH-, -C(=O)O-, -C(=S)O-, -C(=O)S-, -C(=S)S-, -C(=O)NHNH-, -CH2-, -O-, -OC(=O)-, -S-, -SC(=O)-, -NH-, -NHNH-, -NHC(=O)-, -NHNHC(=O)-, -C≡C-, -CH=CH-, a five-membered heterocycle containing at least 2 nitrogen atoms, -CH2C(=O)NH-, -CH2C(=S)O-, -CH2C(=S)S-, -CH2C(=O)NHNH-, -N=CH-, -CH=N-, -NH-N=CH-, and -CH=N-NH-, Y is alkylene C2-C10 chain, R1 is selected from alkyl C1-C46, alkenyl C2-C46, alkynyl C2-C46, Z is selected from H, -OH, -CH3, -CH2OH, -NH2, -C(=O)NH2, -CONH(CH2)2OH, -CON[(CH2)2OH]2, -CONHCH(CH2OH)2, -CONHCH2CH(-OH)CH2OH, -CONH(CH2)2C(=O)NH2, -CON[CH2C(=O)NH2]2, -CONH(CH2)2NHC(=O)NH2, -CONH(CH2)3-N+(CH3)2-(CH2)2-SO3–, -CONH(CH2)3-N+(CH3)2-(CH2)2-COO–, -COO(CH2)2-O-P(=O)(O–)-O(CH2)2-N+(CH3)3, -N+(CH3)2-(CH2)3-SO3–, -N+(CH3)2-(CH2)2-COO-, formula (II), and formula (III), wherein R2 is independently selected from hydrogen and -CH3, E is independently selected from O and S atoms, n is an integer within the range of from 1 to 5, and T is selected from -X-Y-N(R1)2, -C(=O)O(C1-C3 alkyl), -C(=O)OCH2CH2OH, formula (IV), formula (V), formula (VI), -C(=O)OH, -CONH(CH2)2OH, -CON[(CH2)2OH]2, -CONHCH(CH2OH)2, CONH(CH2)2C(=O)NH2, -CON[CH2C(=O)NH2]2, -CONHCH[C(=O)NH2]2, -CONH(CH2)2NHC(=O)NH2, -C(=O)NH2, -CONH(CH2)3-N+(CH3)2-(CH2)2-SO3–, -CONH(CH2)3-N+(CH3)2-(CH2)2-COO–, -NH2, -NHC(=O)CH3, -COO(CH2)2-O-P(=O)(O–)-O(CH2)2-N+(CH3)3, -OH, -O(C1-C3 alkyl), -NHC(=O)NH(CH3), -NHC(=S)N(CH3)2, -NHC(=S)NH(CH3), -NHC(=N-CN)NH2, -NHC(=N-CN)NH(CH3), -NHC(=N-CN)N(CH3)2, -NHC[=N-S(=O)2NH2]NH2, -N+(CH3)2-(CH2)3-SO3–, -N+(CH3)2-(CH2)2-COO–, wherein R2, E and n are as defined above, and/or if Z is -OH or -CH2OH, and T is -C(=O)OH, then Z together with T and three carbon atoms between them may form a cyclic lactone containing 4 to 5 carbon atoms, and pharmaceutically acceptable salts, addition salts and solvates thereof. This lipidoid is useful as a transfection agent. The invention further describes transfection reagents, transfection particles containing this lipidoid, and their use.

Název v anglickém jazyce

Cyclohexane lipidoids for nucleic acid transfection and use thereof

Popis výsledku anglicky

The present invention relates to a lipidoid of general formula I, wherein X is selected from -C(=O)NH-, -C(=O)O-, -C(=S)O-, -C(=O)S-, -C(=S)S-, -C(=O)NHNH-, -CH2-, -O-, -OC(=O)-, -S-, -SC(=O)-, -NH-, -NHNH-, -NHC(=O)-, -NHNHC(=O)-, -C≡C-, -CH=CH-, a five-membered heterocycle containing at least 2 nitrogen atoms, -CH2C(=O)NH-, -CH2C(=S)O-, -CH2C(=S)S-, -CH2C(=O)NHNH-, -N=CH-, -CH=N-, -NH-N=CH-, and -CH=N-NH-, Y is alkylene C2-C10 chain, R1 is selected from alkyl C1-C46, alkenyl C2-C46, alkynyl C2-C46, Z is selected from H, -OH, -CH3, -CH2OH, -NH2, -C(=O)NH2, -CONH(CH2)2OH, -CON[(CH2)2OH]2, -CONHCH(CH2OH)2, -CONHCH2CH(-OH)CH2OH, -CONH(CH2)2C(=O)NH2, -CON[CH2C(=O)NH2]2, -CONH(CH2)2NHC(=O)NH2, -CONH(CH2)3-N+(CH3)2-(CH2)2-SO3–, -CONH(CH2)3-N+(CH3)2-(CH2)2-COO–, -COO(CH2)2-O-P(=O)(O–)-O(CH2)2-N+(CH3)3, -N+(CH3)2-(CH2)3-SO3–, -N+(CH3)2-(CH2)2-COO-, formula (II), and formula (III), wherein R2 is independently selected from hydrogen and -CH3, E is independently selected from O and S atoms, n is an integer within the range of from 1 to 5, and T is selected from -X-Y-N(R1)2, -C(=O)O(C1-C3 alkyl), -C(=O)OCH2CH2OH, formula (IV), formula (V), formula (VI), -C(=O)OH, -CONH(CH2)2OH, -CON[(CH2)2OH]2, -CONHCH(CH2OH)2, CONH(CH2)2C(=O)NH2, -CON[CH2C(=O)NH2]2, -CONHCH[C(=O)NH2]2, -CONH(CH2)2NHC(=O)NH2, -C(=O)NH2, -CONH(CH2)3-N+(CH3)2-(CH2)2-SO3–, -CONH(CH2)3-N+(CH3)2-(CH2)2-COO–, -NH2, -NHC(=O)CH3, -COO(CH2)2-O-P(=O)(O–)-O(CH2)2-N+(CH3)3, -OH, -O(C1-C3 alkyl), -NHC(=O)NH(CH3), -NHC(=S)N(CH3)2, -NHC(=S)NH(CH3), -NHC(=N-CN)NH2, -NHC(=N-CN)NH(CH3), -NHC(=N-CN)N(CH3)2, -NHC[=N-S(=O)2NH2]NH2, -N+(CH3)2-(CH2)3-SO3–, -N+(CH3)2-(CH2)2-COO–, wherein R2, E and n are as defined above, and/or if Z is -OH or -CH2OH, and T is -C(=O)OH, then Z together with T and three carbon atoms between them may form a cyclic lactone containing 4 to 5 carbon atoms, and pharmaceutically acceptable salts, addition salts and solvates thereof. This lipidoid is useful as a transfection agent. The invention further describes transfection reagents, transfection particles containing this lipidoid, and their use.

Druh

P - Patent

CEP obor

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OECD FORD obor

30107 - Medicinal chemistry

Projekt

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Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2025

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Číslo patentu nebo vzoru

AU2022314101

Vydavatel

AU001 -

Název vydavatele

IP Australia

Místo vydání

Phillip ACT

Stát vydání

AU - Austrálie

Datum přijetí

23. 1. 2025

Název vlastníka

Ústav organické chemie a biochemie AV ČR, v. v. i.

Způsob využití

B - Výsledek je využíván orgány státní nebo veřejné správy

Druh možnosti využití

A - K využití výsledku jiným subjektem je vždy nutné nabytí licence