PBAE 456

PBAE 456 is a specific cationic, biodegradable poly(β-amino ester) (PBAE) polymer developed for non-viral gene delivery. Identified through a high-throughput screen of a PBAE library at Johns Hopkins University, PBAE 456 demonstrated superior transfection efficiency in small cell lung cancer (SCLC) cell lines. The polymer self-assembles with plasmid DNA to form nanoparticles that protect the genetic cargo and facilitate its intracellular delivery via endocytosis and subsequent endosomal escape. In preclinical studies, PBAE 456 has been used to deliver wild-type p53 (TP53) to p53-mutant SCLC cells, resulting in p21 induction, cell cycle arrest, and anti-proliferative effects. It has shown potential for both local (intratumoral) and systemic (intravenous) administration in SCLC xenograft models.

Other names

poly(beta-amino ester) 456