Dry Powder Formulation of RNA Nanoparticles for Inhalation and Improved Storage and Transport Conditions

RNhale aims to establish technical and commercial proof-of-concept for our existing and protected protocols to be transformed into a platform technology with the long-term goal of licensing agreements with large pharmaceutical companies which are equipped for continuous manufacturing of large scales of dry powder RNA formulations. RNhale will assess the commercial positioning of a platform technology for spray-drying RNA nanoparticles (NPs). While RNA medicines have revolutionized how we can address a pandemic, RNA therapeutics have not yet broadly changed patient outcomes but are specifically used for very limited indications due to two main reasons: (i) the high immunogenicity and poor stability of RNA; and (ii) the accumulation of nano-encapsulated RNA in the liver and resulting poor targetability of other organs with RNA-loaded NPs. These shortcomings have been addressed in my current ERC project Novel Asthma Therapy by spray-drying polymer-based RNA NPs. RNhale will refine existing protocols for spray-drying RNA NPs made with diverse RNA NPs to overcome current challenges by: 1) improving storage and transport conditions of spray-dried RNA medicines in general; and 2) gaining access to delivery beyond the liver via inhalation delivery to the lung.