Neil Fullarton1, Sarina Saedi1, Kiing Seng Wong1, Christian Witte1 & Elijah Nazarzadeh1
1 Acu-Flow Limited, 255 St Vincent Street, Glasgow, G2 5QY, UK
Summary
Antisense Oligonucleotides (ASOs) present a new paradigm in personalised medicine, facilitating treatments designed to selectively silence disease-driving genes. Inhalation offers a promising non-invasive method of delivering these novel medicines. Key factors, such as high viscosity, shear-induced degradation, and potential for aggregation present major challenges for aerosol delivery and device compatibility. To date, only two ASOs are in development for inhaled delivery. Nebulised delivery may offer a direct non-invasive route to target the lungs more effectively. In this study, we investigate the feasibility of nebulising Danvatirsen, a 16 mer gapmer ASO at clinically relevant dose of 20mg/ml, using Nebu-Flow’s novel technology. Structural and biological integrity of the ASO was confirmed post-nebulisation. These findings support the potential for high-dose ASO delivery via the inhalation route for local delivery to the lungs and treatment of respiratory disorders.