Inhaled antisense oligonucleotide therapies: Inspiration and progress

Inhaled antisense oligonucleotides represent an emerging therapeutic approach for management of respiratory diseases. Although most attention has focused on systemic administration of antisense compounds, this class of agents is currently marketed for local therapy of ocular disease. The inhaled route provides excellent potency in animals due to direct delivery to the target tissue with good tolerability demonstrated in recent clinical trials. The stage is set for the new generation of chemically improved respired antisense oligonucleotides to offer new options for patients with pulmonary disorders.

Section editor:Martin Braddock – AstraZeneca R&D Charnwood, Loughborough, UK