Industry
Clinical trial shows gene editing works for β-Thalassaemia, too
A phase 1 clinical trial published in *Nature* (2026) demonstrated that a transformer base editor can effectively treat severe β-Thalassaemia by modifying patients' own CD34+ haematopoietic stem ce...
A phase 1 clinical trial published in Nature (2026) demonstrated that a transformer base editor can effectively treat severe β-Thalassaemia by modifying patients' own CD34+ haematopoietic stem cells to reactivate fetal haemoglobin (HbF) production. Five patients with severe β-Thalassaemia received a one-time infusion of their own base-edited stem cells, with the editing targeting specific binding sites to reactivate the fetal haemoglobin gene — and all participants ceased regular red blood cell transfusions within one month. Total haemoglobin and HbF concentrations reached 12.4 and 11.5 g/dL respectively at three months post-infusion, and these levels were sustained or improved over a median follow-up period of 23 months.
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Source: industry