Lipids and compositions for intracellular delivery of biologically active compounds

摘要

The present invention provides novel amino-lipids, compositions comprising such amino-lipids and methods of producing them. In addition, lipid nanoparticles comprising the novel amino-lipids and a biologically active compound are provided, as well as methods of production and their use for intracellular drug delivery.

主权项

1. A cyclic amino-lipid of the formula: wherein: R1 is independently selected from
—(CH2)2—N(R)2;—(CH2)2—N(R)—(CH2)2—N(R)2, wherein R is independently selected from —H,C6-40 alkyl, C6-40 alkenyl and C6-C40 alkynyl, provided that —N(R)2 is not NH2;C6-40 alkyl, and C6-40 alkenyl; and R2 is C6-40 alkyl, C6-40 alkenyl or C6-40 alkynyl; and m is 0 or 1, provided that when m is 0, R1 is not C6-C40 alkyl or C6-C40 alkenyl; and pharmaceutically acceptable salts thereof.