Gene therapy

Gene therapy aims to cure thalassaemia using your OWN blood-forming stem cells, so there’s no need to find a matched donor and no risk of graft-versus-host disease. The cells are collected, corrected in the lab, and given back after conditioning treatment.

There are two main approaches: gene addition, which adds a working beta-globin gene using a safe (lentiviral) carrier; and gene editing (such as CRISPR), which switches back on the body’s fetal haemoglobin to compensate. The first products have now been approved in some countries (for example, Zynteglo).

Gene therapy is a major, specialised treatment available at a limited number of centres, and access and cost are still real hurdles — but it’s a genuinely transformative option that’s expanding.