PBAE-447

PBAE-447 is a biodegradable cationic poly(β-amino ester) (PBAE) polymer designed for high-efficiency, low-toxicity non-viral gene delivery. Synthesized via Michael addition and end-capping, it is used to encapsulate genetic material, such as plasmid DNA, into nanoparticles. This delivery platform is particularly notable for its ability to overcome transfection barriers in hard-to-transfect cells, such as non-activated primary T cells, which are critical for advanced immunotherapies like CAR-T. By integrating microfluidic co-assembly with PEG or PEG-lipid functionalization (e.g., DMG-PEG2000), PBAE-447 nanoparticles achieve enhanced serum stability and sustained gene expression. The platform aims to simplify CAR-T manufacturing by eliminating the need for in vitro T cell activation and holds potential for direct in vivo gene therapy applications.

Other names

Poly(beta-amino ester) 447