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US 2019/0314511 A1Published application
Optimized in vivo delivery system with endosomolytic agents for nucleic acid conjugates
AssigneesOnxeo, Institut Curie, Centre National De La Recherche Scientifique
InventorsJIAN-SHENG SUN, MARIE DUTREIX, MARIA QUANZ
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Abstract
The present invention relates to an optimized in vivo delivery system with endosomolytic agents for nucleic acid of therapeutic interest conjugated to molecules facilitating endocytosis, in particular for use in the treatment of cancer.
Claims14
A conjugated hairpin nucleic acid molecule which has the following formula:
- wherein N is a nucleotide, n is an integer from 15 to 195, the underlined N refers to a nucleotide having or not a modified phosphodiester backbone, L′ is a linker;
- m is 1 and L is a linker, C is a molecule facilitating endocytosis selected from the group consisting of single or double chain fatty acids, tocopherol, folates or folic acid, cholesterol, sugar, peptide and protein, and wherein C is conjugated via L to the linker L′.
Dependencies are inferred from the claim text.