The first drug to treat two genetic blood disorders has been approved by the UK Medicines Agency
Britain has approved a gene therapy aimed at treating sickle cell anemia and another inherited blood disorder for patients aged 12 and over, the country’s medicines agency said today, 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 earned its inventors Emmanuelle Charpentier of France and Jennifer Doudna of the United States the 2020 Nobel Prize in Chemistry, Britain’s Medicines and Healthcare Products Regulatory Agency said and Healthcare products Regulatory Agency, MHRA).
THE sickle cell anemia and β-thalassemia are genetic conditions caused by errors in the genes for hemoglobin, which red blood cells use to carry oxygen around the body.
“Both sickle cell disease and beta-thalassemia are painful ailments, which last a lifetime and which in some cases can be fatalMHRA interim director Julian Beach said in the statement.
In clinical trials Casgevy was found to restores healthy hemoglobin production in the majority of participants with sickle cell disease and transfusion-dependent beta-thalassemia, alleviating the 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.
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