Apriligen Sweden, based in Lund, is the European research and development centre of Apriligen, a clinical-stage gene therapy company focused on rare monogenic blood diseases in children. The lead programme, APR-2020, is a one-time autologous lentiviral gene therapy for Diamond-Blackfan anemia caused by RPS19 deficiency, now in a registrational clinical study in the United States. Building on stem cell and gene therapy research from Lund University, the Swedish team drives research, process and analytical development, intellectual property and the preparation of European manufacturing and clinical activities. Apriligen also develops small-molecule CDK8/19 inhibitors for the disease. The goal is a cure for patients who today depend on lifelong transfusions or stem cell transplantation.
