EU approves first gene therapy for patients with a mild form of β-Thalassemia

Exciting news in the personalised medicine space – the EU has just approved the first gene therapy for patients with a mild form of β-Thalassemia.  Patients with transfusion-dependent β-thalassemia (TDT) have mutations in the β-globin gene that mean that they have reduced levels of haemoglobin, the protein that carries oxygen around the blood. 

The treatment works by inserting a functional form of the β-globin gene into a patient’s own blood stem cells.  The stem cells are then returned to the patient, where they produce blood cells containing functional haemoglobin.  This treatment offers long-term change for these patients, who’ve previously been reliant on regular blood transfusions.  Interestingly, the EU has approved the drug specifically for patients 12 years or older, and who do not have the β0/β0 genotype – exactly the kind of defined patient subgroup that can contribute to the novelty and inventiveness of a second medical use claim before the EPO. 

You can read more about this development here.