(260) Reproducible and scalable polymer nanoparticle manufacturing facilitates clinical translation

Introduction: Polymer nanoparticles (PNPs) are a promising delivery method for gene therapies. PNPs can overcome known concerns for AAVs and LNPs, such as cargo capacity limitations, adverse immune responses, cell-based manufacturing challenges, and high cost of scaled production [1]. Reproducibility is crucial to successful delivery vehicle discovery and enables increased production to support clinical translation. This work used a rapid, iterative PNP discovery platform to demonstrate progress toward reproducible and scalable PNP manufacturing intended to facilitate gene therapy clinical translation.