Project description
Inhaled RNA therapy to improve targeted drug delivery
Conventional RNA drugs can be used only for limited indications owing to the poor RNA stability and high immunogenicity (the ability of the antigen to provoke an immune response in the body). Furthermore, RNA nanoparticles accumulate in the liver; therefore, targeted delivery to other organs is not satisfying. The EU-funded RNhale project will seek to overcome these drawbacks by developing RNA nanocarriers that can be stabilised by means of spray drying, producing a dry powder. The project will advance the targeted delivery of the powder through inhalation and improve storage and transport by optimising the spray-drying protocols.
Objective
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.
Fields of science
Programme(s)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Funding Scheme
HORIZON-AG-LS - HORIZON Lump Sum GrantHost institution
80539 MUNCHEN
Germany