Alexion Pharmaceuticals, Inc.
BIODEGRADABLE ACTIVATED POLYMERS FOR THERAPEUTIC DELIVERY

Abstract:

Activated polymers comprising one or more backbone ester(s) are disclosed. In particular, activated poly(amine-co-ester) (aPACE) terpolymers and methods of making and using these aPACE terpolymers are disclosed. These aPACE terpolymers can be used to safely and efficiently deliver biomolecules, in particular nucleic acids, to cells, both in vitro and in vivo. Methods for making activated polymers are also provided. Furthermore, methods for delivering mRNA and methods of gene therapy using activated polymers, in vitro and/or in vivo are further disclosed.