申请人:National University Corporation
Tokyo Medical and Dental University
公开号:EP3770257A1
公开(公告)日:2021-01-27
The object of the present invention is to provide a nucleic acid agent that can be efficiently delivered to the nervous system, particularly the central nervous system to which the BBB mechanism prevents drug delivery, and can produce an antisense effect on a target transcriptional product at the delivered site, and a composition comprising the same.
Provided is a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand capable of hybridizing to part of a target transcriptional product, and has an antisense effect on the target transcriptional product, to a second nucleic acid strand comprising a base sequence complementary to the first nucleic acid strand, and is bound to tocopherol or an analog thereof, cholesterol or an analog thereof, or a substituted or unsubstituted C1-30 alkyl group, a substituted or unsubstituted C2-30 alkenyl group, or a substituted or unsubstituted C1-30 alkoxy group.
本发明的目的是提供一种核酸制剂,它可以有效地递送到神经系统,特别是BBB机制阻碍药物递送的中枢神经系统,并能在递送部位对靶转录产物产生反义效应,以及包含该制剂的组合物。
本发明提供了一种双链核酸复合物,该复合物通过将能够与目标转录产物的一部分杂交的第一核酸链与包含与第一核酸链互补的碱基序列的第二核酸链退火形成,并对目标转录产物具有反义作用、并与生育酚或其类似物、胆固醇或其类似物、或取代或未取代的 C1-30 烷基、取代或未取代的 C2-30 烯基、或取代或未取代的 C1-30 烷氧基结合。