CRISPR therapy frees Berlin teen with beta-thalassaemia from transfusions

By Adrian Cole · October 1, 2026, 8:34 PM · 3 mins read

A 19-year-old in Berlin with beta-thalassaemia no longer needs blood transfusions after getting Casgevy, the first CRISPR gene-editing drug used in Germany.

Mohammad, 19, spent years tied to blood transfusions. That changed in Berlin. He became the first person in Germany with beta-thalassaemia to get Casgevy, a CRISPR gene-editing therapy, at Charité hospital. This marks a real shift for gene-editing medicine in the country.

Casgevy has been approved in the European Union since 2024 for patients aged 12 and up with sickle cell disease or beta-thalassaemia. The drug targets the faulty haemoglobin gene at the root of the disorder. Beta-thalassaemia causes severe tiredness, pain, stunted growth, and dangerous iron buildup. Each year, about 60,000 children worldwide are born with severe forms. Many need transfusions every three weeks. These transfusions bring their own risks over time.

During the CRISPR therapy at Charité, Mohammad received more than 900 million genetically modified cells after preparatory chemotherapy.

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Gene-editing opens a new door

Stem cell transplants are the standard cure but are usually only for patients under 14. Mohammad was too old for this. In May 2026, he became the first in Germany to get Exagamglogene Autotemcel, a CRISPR-based treatment, at Charité. The process takes about a year. The result is clear: Mohammad no longer needs transfusions. His immune system has bounced back. Charité says he is doing very well.

The technology behind this comes from Nobel winners Emmanuelle Charpentier and Jennifer Doudna. CRISPR lets doctors edit the faulty gene with precision. The treatment is complex and resource-heavy, so only a few patients will get it for now. But for those who do, the impact is huge. According to a detailed report by RND, Charité is the first certified center in Germany to offer this therapy. Statutory health insurance covers the cost if the clinic gives special approval.

Barriers and questions remain

Some experts are still cautious. The process is tough, and only a small group of patients can get the therapy. Long-term effects are not yet known. More evidence and close follow-up are needed. This is similar to what happened with other advanced treatments, like the single-cell brain atlas project. Big breakthroughs often raise new questions about who can benefit and how widely.

The full course of Casgevy therapy at Charité is designed to last about 12 months, and after the procedure, Mohammad's immune system has already recovered with his condition described as very good.

Tagesspiegel

Gene-editing is now part of German clinical care for inherited blood disorders. A young adult who once had no curative options is now free from transfusions. That is a real change for rare disease medicine. But the bigger test is whether this kind of therapy can reach more people. For now, Berlin shows what CRISPR can do. The promise is real, but access is still limited.

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Adrian Cole

Adrian Cole is the Founder and Editor-in-Chief of GenoMethods, where he writes about bioengineering, genome and cell engineering, synthetic biology, computational biology and emerging research methods. His editorial approach focuses on how technologies actually work, how they are validated and where the evidence stops supporting the claim.