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Posted on 04 June 2019

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.

Read more about precision medicine in our dedicated blog series.

Frances Salisbury

Contact Frances Salisbury

Fran is a member of our life sciences patent team with expertise in the biotechnology, bioinformatics, pharmaceutical and agricultural science sectors. Fran prepares and prosecutes applications relating to a range of precision medicine inventions. She handles a number of applications relating to biomarkers and diagnostics, including methods for the identification of biomarkers and biomarker panels, and their use in diagnosing and prognosing disease. Fran enjoys the particular challenges that prosecution of these cases around the world brings, arising from the divergent approaches taken by the different patent offices. Her clients include universities and research organisations, start-ups, spin outs and multi-national companies. She has a degree in Biological Sciences from the University of Bristol and a PhD in molecular plant genetics is from the University of Edinburgh.

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