New vehicles to bring RNA to its site of action
RNAs hold enormous potential as therapeutics, yet their translation into medicines critically depends on delivery vehicles to package the RNA and bring it to its site of action. In this project three RNA Biotech companies and two universities will join forces to advance targeted RNA delivery.
In total 6-8 % of the population suffers from one of the about 6000 rare diseases creating an enormous societal burden. For many of these, RNA has a potential as medicine. RNAs (mRNA, siRNA, antisense RNA) have well understood working principles enabling a rational and rapid design of therapeutic modalities. Considering the recent success of the first RNA drugs, it can be expected that RNAs will change the ways how medicines are developed, thereby providing therapies also for rare diseases at affordable costs.
In this project, two universities and three RNA biotech companies (siRNA services, mRNA therapeutics, mRNA production and formulation) will join forces to develop lipid nanoparticles for targeted RNA delivery. The innovative aspect is the use of a lipid nanoparticle formulation that shows less preference for the liver and thereby should enable better targeting to other organs through functionalisation with targeting ligands. Targeted delivery will be investigated in animal models and in innovative microfluidic organ-on-a-chip systems.
The project will deliver protocols and proof-of-concepts for the formulation of different types of RNA (mRNA, siRNA, antisense RNA) and coupling of targeting ligands for cellular receptors on tumor cells and other types of tissues. For the biotech companies this will directly translate into new marketable products, for the universities into the strengthening of research lines towards therapy development. Overall, the project results will contribute to making affordable RNA-based therapeutics a reality for patients with rare diseases.