To facilitate full-body AON treatment in the future, we are currently developing AON formulations that allow a systemic delivery method that is safe, tolerable and efficient in reaching all skeletal and cardiac muscles. We have therefore set up different lines of preclinical research :
1. Identification of muscle-homing ligands that enhance the uptake of AON molecules from the blood circulation by the muscle fibers .
2. Identification of drug carriers that enhance the uptake AON molecules through the extracellular matrix of muscle fibers.
3. Assessment of compound distribution, delivery efficiencies, safety parameters, and improved muscle performance in mdx mice.
4. Application of optimal formulation to human sequence-specific AONs in hDMD mouse model(s)