Lipocationic polymers and uses thereof

摘要

Polymers produced by ring opening polymerization which comprises an amino group that can be used in compositions to deliver a nucleic acid such as a miRNA or a siRNA. In some embodiments, compositions which comprise the polymers described herein and a nucleic acid are also provided herein. In some embodiments, these compositions are used to silence one or more genes in vivo or treat a disease or disorder.

主权项

1. A polymer comprising a formula: wherein:
X1 is alkyl(C≦18) or substituted alkyl(C≦18);X2 is hydrogen, alkyl(C≦18), or substituted alkyl(C≦18);R1 is -A-Z;wherein:
A is an alkanediyl(C≦18) or substituted alkanediyl(C≦18);Z is —NR3R4;wherein:
R3 and R4 are each independently selected from hydrogen, alkyl(C≦18), substituted alkyl(C≦18); or R3 and R4 are taken together and are alkanediyl(C≦18) or substituted alkanediyl(C≦18);R2 is alkyl(C≦24), alkenyl(C≦24), substituted alkyl(C≦24), or substituted alkenyl(C≦24);x and y are each independently 0, 1, 2, 3, 4, or 5;m and n are each independently an integer between 0 and 250, provided that at least one of m and n is greater than 1; andthe repeating units defined by m and n are randomly distributed throughout the polymer; or a pharmaceutically acceptable salt thereof.