Camille Jones
BME PhD Proposal Presentation

Date: 2026-10-28
Time: 12:00pm
Location / Meeting Link: HSRB I Auditorium / https://emory.zoom.us/j/97696330685

Committee Members:
James Dahlman, PhD (Advisor); Hyejin Kim, PhD; Felipe Quiroz, PhD; Simone Douglas-Green, PhD; M.G. Finn, PhD


Title: Clinically relevant lipid nanoparticles for pulmonary delivery 

Abstract:
Lipid nanoparticles (LNPs) are used to deliver FDA-approved vaccines and hepatic therapies. However, no FDA-approved RNA-LNP therapies target the lung. Recent mRNA-LNP clinical trials for cystic fibrosis have failed due to tolerability and efficacy issues and were administered via inhalation. Systemic lung delivery avoids many of the physical barriers in diseased lung states that limit inhaled therapies, but clearance by the liver causes systemically administered LNPs to accumulate there. Incorporating positively charged components is the traditional way to achieve systemic pulmonary delivery of RNA-LNPs and reduce liver delivery. Although effective for developing lung-targeting LNPs, positively charged lipids can be toxic. In this work, I propose developing and characterizing safe, efficacious positively charged LNP formulations that target lung epithelial cells. First, I engineered LNP formulations optimized for pulmonary delivery in mice. Next, I characterized the physicochemical properties and safety profiles that govern the clinical translatability of pulmonary LNPs. Finally, I evaluated the biodistribution, functional delivery, and tolerability of optimized pulmonary LNP formulations in end-of-life non-human primate models. This work presents preclinical data for a novel pulmonary-targeted LNP, providing the field with a clinically relevant intravenous delivery vehicle for a myriad of pulmonary diseases.