Charité Berlin administers first CRISPR gene therapy in Germany

A 19-year-old patient at Charité – Universitätsmedizin Berlin has become the first person in Germany to receive CRISPR-based gene therapy as part of regular medical care. The patient, who suffers from severe beta-thalassemia, was treated in May with Exagamglogene Autotemcel (brand name Casgevy).

The therapy utilizes CRISPR/Cas9 technology to genetically modify the patient’s own blood-forming stem cells in a laboratory. By altering a regulatory region of the BCL11A gene, the cells are prompted to produce fetal hemoglobin, which can compensate for the defective adult hemoglobin caused by the disease. Following the infusion of approximately 900 million modified cells and a necessary chemotherapy conditioning process, the patient has reportedly no longer required regular blood transfusions four months after treatment.

Casgevy has been conditionally approved in the European Union since 2024 for patients aged 12 and older with transfusion-dependent beta-thalassemia or sickle cell disease. While the treatment offers significant potential, medical professionals note that long-term safety data is still being gathered, and patients are subject to a 15-year follow-up period. The conditioning process required for the therapy carries risks, including potential infertility and damage to the liver or mucous membranes.