申请人:Arbutus Biopharma Corporation
公开号:EP3156077A1
公开(公告)日:2017-04-19
The present invention provides targeting lipids of structure (Cl) L100 - linker - L101, where L100 is a lipid, lipophile, alkyl, alkenyl or alkynyl, L101 is a ligand or -CH2CH2(OCH2CH2)pO(CH2)qCH2-ligand, p is 1-1000, and q is 1-20. In addition, the invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo.
本发明提供了结构为(Cl)L100-连接体-L101的靶向脂质,其中L100为脂质、亲脂质、烷基、烯基或炔基,L101为配体或-CH2CH2(OCH2CH2)pO(CH2)qCH2-配体,p为1-1000,q为1-20。此外,本发明还提供了向细胞输送治疗剂的组合物和方法。其中特别包括新型脂质和核酸-脂质颗粒,它们能有效地封装核酸,并在体内将封装的核酸有效地递送到细胞中。