At Glafabra Therapeutics we use patient cells to deliver a gene therapy and create patient freedom from the tedium of traditional ERT (enzyme replacement therapy). We harvest patient cells and genetically-engineer them to express a missing enzyme. Reintroduction of the modified cells back into the patient achieves a therapy that can last for 5 years after a single infusion. Glafabra is launching plans to apply their successful approach to a wide variety of metabolic disorders.