Pompe disease is a rare disease affecting approximately one in 40,000 people worldwide. There are currently no cures for the disease, but enzyme replacement therapy (ERT) has recently been introduced as the most effective option. Current ERT therapies have high rate of adverse events, as well as risk of severe allergic reactions, immune-mediated reactions and cardiorespiratory failure. Other limitations include a short half-life in the bloodstream, poor uptake and the requirement for repeated dosing (every two weeks). Currently, ERT costs up to $500,000 per patient per year, an estimated market of $700M per year. The current market for Pompe disease is predicted to grow at a rate of 11.5% between 2016 and 2020.