Charité Berlin treats first patient in Germany with commercial CRISPR gene therapy

First treatment in standard German healthcare

Doctors at Charité Universitätsmedizin Berlin have treated a patient using CRISPR gene editing outside clinical trials for the first time in Germany. The 19-year-old patient, named Mohammad, received the gene therapy Exagamglogene Autotemcel, commercially known as Casgevy, on 28 May 2026 to treat severe beta-thalassemia. Four months after receiving the infusion, the university hospital reported that the patient no longer requires blood transfusions and has returned to normal daily routines. Emmanuelle Charpentier, who co-discovered the CRISPR-Cas9 mechanism alongside Jennifer Doudna in 2012, was present at Charité during the administration. The European Union granted conditional authorization for the drug in 2024 for patients aged 12 and older suffering from transfusion-dependent beta-thalassemia or severe sickle cell disease. Charité is the first medical centre in Germany qualified to administer the specialized therapy.

Genetic disorder and treatment limits

Beta-thalassemia is an inherited blood disorder caused by genetic mutations that prevent the body from producing functional hemoglobin, the iron-rich protein that transports oxygen in red blood cells. Around 60,000 children worldwide are born with the severe form annually, facing severe fatigue, pain, physical and cognitive delays, and early death if left untreated. Patients traditionally require donor blood transfusions every three weeks, a regimen that causes progressive iron accumulation and subsequent organ damage over time. Conventional allogeneic stem cell transplants from matched donors can cure the condition, but doctors limit the procedure to patients under 14 years of age due to escalating complication risks. Mohammad had passed that age threshold, leading his statutory health insurance provider to approve the gene therapy after an individual case review. Charité senior physician Lena Oevermann described the transition from laboratory research to standard clinical practice.

This has turned basic research into medical care.

Laboratory editing and stem cell infusion

The treatment protocol developed by Vertex Pharmaceuticals and CRISPR Therapeutics spanned approximately 12 months. Medical staff initially extracted blood stem cells from the patient and shipped them to manufacturer laboratories in the Netherlands for genetic modification. Technicians used the CRISPR tool to reactivate the gene responsible for the gamma-globin chain, restoring the synthesis of fetal hemoglobin. Mohammad then underwent a cycle of high-dose chemotherapy designed to eliminate existing marrow cells and create physical space in the bone marrow. On 28 May 2026, clinicians infused more than 900 million edited stem cells back into his bloodstream.

Post-infusion recovery and mandatory monitoring

Mohammad remained in inpatient hospital care for nearly six weeks following the infusion before discharge. Laboratory assessments show that fetal hemoglobin now constitutes 85% of his total hemoglobin, bringing his overall blood count into the normal range. His immune system has recovered, and hospital officials stated that he plans to begin vocational training. Oevermann pointed out that the preparatory high-dose chemotherapy involves risks, including painful mucosal inflammation, potential liver toxicity, and a high probability of infertility. Because European regulators approved the drug conditionally, medical teams must track Mohammad and other recipients for 15 years to collect data on long-term safety and therapeutic durability.