Hybrid extracellular vesicles for advanced treatment of lysosomal storage diseases

HEAL aims to transform the treatment of lysosomal storage disorders, focusing on Fabry disease, through third-generation nanoparticles that combine extracellular vesicles and liposomes (EVs) to deliver highly active enzymes to target organs. The project integrates artificial intelligence to optimise design, targeting and scalable manufacturing, and evaluates efficacy in advanced preclinical models that better replicate human disease. The goal is to improve stability, biodistribution and potential access to the brain, overcoming the limitations of conventional ERT.