Inhaled Nucleic Acid Therapeutics and Biopharmaceuticals


DDL Emerging Scientist Award 2020:

Research Synopsis Inhaled Nucleic Acid Therapeutics and Biopharmaceuticals


Since my PhD study, I have been working in the field of non-viral gene delivery. The use of nucleic acids to manipulate gene expression is a powerful therapeutic strategy for the treatment of many diseases. A safe and effective delivery system in the key for the successful translation of nucleic acid therapy from bench to bedside. I entered the field of pulmonary drug delivery in 2010 when I noticed that there is a gap of research in the development of inhaled delivery system for nucleic acids, especially RNA therapeutics [1]. Majority of the work in the field of RNA delivery has been focused on systemic administration of nanoparticles by injection which often has a poor penetration to the lung. RNAs have huge therapeutic potential for the treatment of a wide range of lung diseases including severe asthma, cystic fibrosis, viral infections and lung cancer, but the lack of an efficient inhaled RNA delivery system has hindered their clinical application in this important disease area. Since then, I have been focused on the development of inhaled powder formulation of nucleic acid therapeutics. With the recent FDA approval of siRNA therapeutics (patisiran and givosiran), both of which target hepatic disorders, the field of RNA therapy is ready to look for applications beyond the liver.