MHRA approves first ‘gene-editing’ treatment for sickle-cell disease and thalassemia

It can restore healthy haemoglobin production in patients and free them from painful symptoms  

The Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised an innovative and first-of-its-kind gene-editing treatment for sickle-cell disease and transfusion-dependent β-thalassemia.

Known as Casgevy (exagamglogene autotemcel), the treatment is based on the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

Casgevy is the first medicine to be licensed that uses CRISPR, and it is to be used for treating patients aged 12 and over.

To date, a bone marrow transplant has been the only permanent treatment option for these life-long conditions, which in some cases can be fatal.

The MHRA’s authorisation came after a rigorous assessment of its safety, quality and effectiveness.

Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA, informed that Casgevy has been found to “restore healthy haemoglobin production” in the majority of study participants, relieving their symptoms.

“The MHRA will continue to closely monitor the safety and effectiveness of Casgevy, through real-world safety data and post-authorisation safety studies being carried out by the manufacturer,” he said.

How the gene therapy work?

Both sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, a protein used by red blood cells to carry oxygen around the body.

This genetic error can lead to attacks of very severe pain, serious and life-threatening infections, and anaemia in people with sickle cell disease.

People with β-thalassaemia can experience severe anaemia, and they may need a blood transfusion every 3 to 5 weeks, and injections and medicines throughout their lives.

Casgevy is designed to treat these conditions by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

First, stem cells are taken out of the patient’s bone marrow, edited in a laboratory and then infused back into the patient.

As stated by the MHRA, the results of this treatment have the potential to be life-long, which is good news for patients suffering from these genetic conditions.