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UK gives ‘green light’ to gene therapy aimed at curing sickle cell disease

“Yes” to genetics treatment which aims to treat sickle cell disease anemia and another inherited blood disorder for patients aged 12 and over, Britain said on Thursday (16/11) as the country’s medicines agency reported, making it the first country in the world to do so.

Casgevy is the first drug to be licensed using the CRISR genome editing tool, which won its inventors Emmanuelle Charpentier of France and Jennifer Doudna of the United States the 2020 Nobel Prize in Chemistry, the British body said medicines and health products (Medicines and Healthcare products Regulatory Agency, MHRA).

What is sickle cell anemia and beta-thalassemia

Sickle cell anemia and β-thalassemia are genetic diseases caused by errors in the genes for hemoglobin, which red blood cells use to carry oxygen around the body.

“Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal”the interim director of the MHRA, Julian Beach, said in the statement as reported by the Athens News Agency citing Reuters.

In clinical trials, Casgevy was found to restore healthy hemoglobin production in the majority of participants with sickle cell disease and transfusion-dependent beta-thalassemia, alleviating symptoms of the disease, Beach added.

The MHRA said no major safety concerns had been identified during the trials, adding that it was closely monitoring the safety of the medicine.

The treatment consists of removing primordial hematopoietic cells from the patient’s bone marrow and correcting a gene in the laboratory, with the modified cells reintroduced into the patient’s body after adaptation therapy to prepare the bone marrow.

Source: News Beast

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