Enzyme replacement therapy and cost savings
For the treatment of Pompe disease and Hunter syndrome, patients currently receive so-called enzyme replacement therapy. This treatment is extremely expensive, costing hundreds of thousands of euros per patient each year. We are developing our lentiviral gene therapy with the goal that, after a single treatment, this costly enzyme replacement therapy can be largely or even entirely replaced. This would lead to substantial savings in healthcare costs.
Preparing for future gene therapies
This approach enables us to move on to developing gene therapies for diseases for which no treatment currently exists. By that time, it is crucial that we already have extensive knowledge, data, and infrastructure in place—and that cost savings have been achieved—so that we can keep the costs of future therapies as low as possible.
Societal distribution of costs
For many of these diseases, society also incurs significant long-term costs to support patients throughout their lives. However, these costs are often covered by budgets other than those allocated for medicines and treatments.
Medical and scientific advantages
There is also a strong medical and scientific rationale. Erasmus MC is home to the world’s leading center of expertise for Pompe disease, knowledge that is invaluable for developing gene therapy for this condition. In addition, all patients in the Netherlands with Hunter syndrome are treated at Erasmus MC, making it a logical second disease for which to develop a therapy.
