AerosoLNP Lung-targeted Delivery Platform

Background

Alpha 1 Antitrypsin ( A1ATD) disease is estimated to affect 70,000 – 100,000 people in the US. Treatment options for this disease are focused on maintenance of lung function and infection prevention. There is an unmet need for therapeutics that provide improvements in lung function for A1ATD.

Delivery of mRNA and gene editing cargo is a current area of intense research. Ex vivo approaches are fraught with severe complications, including the requirement for the patient to undergo chemotherapy which carries the risk of infertility. In vivo delivery of cargo is currently limited to intramuscular delivery to prime the immune system or intravenous delivery targeting the liver.

Technology Overview

Researchers at The University of Texas at Austin (UT Austin) have developed a lung epithelial cell targeted, aerosolizable lipid nanoparticle formulation that penetrates lung mucus to deliver cargo. This lung delivery platform has applications for disease treatment and vaccine delivery. This technology has been demonstrated to effectively deliver a reporter mRNA. The technology is being further developed through the UT Austin Life Science Technology Development Program for treating A1ATD where the lungs are subject to serine protease attacks that trigger chronic obstructive pulmonary disease (COPD) and cause lung dysfunction.

Stage of Development

This technology is currently being developed by the UT Austin Life Science Technology Development Program, with studies using an animal model of A1ATD underway.

Opportunity

UT Austin is seeking partners to participate in development of the AerosoLNP lung delivery platform.