Nucleic acid-based therapy has recently been established as a new class of drugs with the

the capacity to both up- and downregulate gene expression. The programmable nature of RNA drugs provides them with almost unlimited potential for treatment of human diseases. However, many hurdles must still be overcome before the full capacity of RNA drugs can flourish. The overall aim of RNA-META is to conduct innovative basic and preclinical research within the area of nucleic acid-based therapeutics and translate our most promising results into viable new therapies. We will focus on solving some of the main challenges that remain before RNA therapeutics can be widely implemented in clinical programs namely:

• Enhancing the function and efficiency of RNA therapeutics at the molecular level through chemico-biological engineering.
• Targeting of specific cells and tissues relevant for treating metabolic diseases
• Improving bioavailability by optimizing the delivery route both outside and inside cells

We believe that advancements in these areas will significantly lower the required treatment dose and make RNA therapeutics effective, scalable, affordable, and safe for the patient.