Hemophilia is a severe bleeding disorder caused by a deficiency of Factor VIII or IX. Patients are treated with intravenously administered coagulation factor concentrate 2-4 times every week to prevent severe and frequent bleeding episodes

AAV-based gene therapy aims to increase factor levels in order to reduce the risk of bleeding by expression of FVIII and IX in the liver. In the Erasmus MC 6 patients have successfully been treated in the last few years, leading to reduced bleeding without the need of factor concentrates.

In this webinar we will report the latest results on efficacy, side effects and hurdles of gene therapy. Given the increased number of gene therapy initiatives for other rare diseases, the Erasmus MC should invest in gene therapy in order to cure rare inherited disorders and improve patient outcome and quality of life.

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