July 13, 2015
The ability to control chemical functionality is an exciting feature of modern polymer science that enables precise design of drug delivery systems. Ring-opening polymerization of functional monomers has emerged as a versatile method to prepare clinically translatable degradable polyesters.1 A variety of functional groups have been introduced into lactones; however, the direct polymerization of tertiary amine functionalized cyclic esters has remained elusive. We report a strategy that enabled the rapid synthesis of >130 lipocationic polyesters directly from functional monomers without protecting groups. These polymers are highly effective for siRNA delivery at low doses in vitro and in vivo.
Publication - Abstract
Jan 30, 2020
Advanced Materials
Publication - Abstract
Aug 18, 2020
Nucleic Acids Research
In this paper Alnylam Pharmaceuticals, developer of RNAi therapeutics ONPATTRO® and GIVLAARI®, investigates the biological origins of the extended duration of N-acetylgalactosamine silencing RNA (GalNAc-siRNA) activity observed in vivo.