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公开(公告)号:US20190298859A1
公开(公告)日:2019-10-03
申请号:US16337243
申请日:2017-09-27
Applicant: UCB Biopharma SPRL
Inventor: Terence Seward Baker , Michael Anthony William Eaton , Timothy John Norman , James Petrie Turner
Abstract: Compounds of formula (I), wherein Ar is an aryl group optionally further substituted with one or more groups R3; A is a lipophilic, hydrophobic moiety; R1 is a phosphodiester, phosphotriester, thioether or amide group; X is an unsubstituted or substituted C6 to C24 alkylene or alkenylene group, which is optionally interrupted by one or more —NR9—, —O— or —S— linkages, R2 is —YC(R4)(R5)CO2R6; and pharmaceutically acceptable salts or solvates thereof are useful as endosomolytic agents particularly for the delivery of nucleic acids useful in gene therapy.