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U.S. approves two ‘breakthrough’ gene therapies for sickle cell treatment

The United States Food and Drug Administration (FDA) has, on Friday, approved a therapy called Casgevy made by Vertex Pharmaceuticals and CRISPR Therapeutics for the treatment of sickle cell disease.

According to NBC News, it is the first medicine that uses the gene-editing tool CRISPR to be approved for treatment of sickle cell in the United States. In 2020, the investors of the treatment method won the Nobel Prize in chemistry.

Sickle cell disease is an hereditary blood disorder passed from parents to their children that reduces the lifespan of the red blood cells, causing anemia with more than 120 million people suffering from it worldwide.

According to the World Health Organisation (WHO), more than 66 per cent of sickle cell patients live in Africa. About 1,000 children born daily in the region carry the disease, making it the most prevalent genetically-acquired disease in the region.

Also, the FDA approved another sickle cell disease treatment called Lyfgenia, a gene therapy produced by drugmaker Bluebird Bio. The two approved treatments work by genetically modifying a patient’s own stem cells, NBC reported.

Until now, bone marrow transplant was the only method for treatment of sickle cell disease but the treatment is a difficult process due to risk of rejection by the immune system of the recipient and the scarcity when looking for a matching donor.

“I think this is a pivotal moment in the field,” Dr Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, who has previously consulted for Vertex, was quoted by NBC.

He added: “It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product.”