26-01-2017 дата публикации
Номер: US20170021036A1
Принадлежит:
The present invention relates to oligomers, polymers and lipidoids comprising characteristic oligo(alkylene amine) moieties which are useful as vehicles for transfecting a cell with RNA. The present invention furthermore relates to a composition comprising at least a nucleic acid and an oligomer or polymer or a lipidoid comprising such oligo(alkylene amine) moieties and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses. 2. The composition of claim 1 , comprising the RNA and the component comprising an oligo(alkylene amine) selected from components a) and b) claim 1 , wherein: {'br': None, 'sup': 2', '3', '4', '5', '6, 'sub': 2', '2', 'a', '2', '2', 'b', 'm', '2', '2', 'a', 'n, '—NR{CH—(CH)—NR—CH—(CH)—NR}—[CH—(CH)—NR]—R\u2003\u2003(IIa),'}, 'component a) is an oligomer or polymer comprising a plurality of groups of formula (IIa) as a side chain and/or as a terminal group{'sup': 2', '6, 'claim-ref': {'@idref': 'CLM-00001', 'claim 1'}, 'wherein a, b, m, n, and Rto Rare defined as in , and wherein one or more of the nitrogen atoms indicated in formula (IIa) may be protonated to provide a cationic oligomer or polymer structure; and'} {'br': None, 'sup': 2', '3', '4', '4', '6, 'sub': 2', '2', 'a', '2', '2', 'b', 'm', '2', '2', 'a', 'n, '—NR{CH—(CH)—NR—CH—(CH)—NR}—[CH—(CH)—NR]—R\u2003\u2003(IIIa),'}, 'component b) is an oligomer or polymer comprising a plurality of groups of formula (IIIa) as repeating units{'sup': 2', '5, 'claim-ref': {'@idref': 'CLM-00001', 'claim 1'}, 'wherein a, b, m, n, and Rto Rare defined as in , and wherein one or more of the nitrogen atoms indicated in formula (IIIa) may be protonated to provide a cationic oligomer or polymer structure.'}3. The composition of claim 1 , comprising the RNA and a lipidoid having the structure of formula (IVa):{'br': None, 'sup': 1', '2', '3', '4', '5', '6, 'sub': 2', '2', 'a', '2', '2', 'b', 'm', '2', '2', 'a', 'n, 'R—NR{CH—( ...
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