FDA approves CRISPR-based gene therapy for beta thalassemia » Medvestnik

The FDA has approved Casgevy for the treatment of beta thalassemia. The cost of administering the drug will be $2.2 million.

The US Food and Drug Administration (FDA) has approved Casgevy, a drug based on CRISPR/Cas9 genome editing technology, for the treatment of beta thalassemia. This was reported by the Pharmaceutical Bulletin with reference to the company Vertex Pharmaceuticals, which developed gene therapy together with CRISPR Therapeutics.

This is the second indication for Casgevy registered by the US regulator. In December, the FDA approved the drug as a treatment for sickle cell disease, making it the first CRISPR-based treatment to be approved.

Introduction Casgevy is a complex process that involves extracting human stem cells and editing their genes using CRISPR technology in a specialized laboratory. The edited genes are returned to the patients’ bodies. The drug works against both sickle cell anemia and thalassemia, since both diseases are caused by an error in the genes responsible for hemoglobin synthesis.

Vertex said it is working to open a network of dispensaries in the United States where patients can receive the drug. Today, gene therapy can be administered into the body only in nine authorized medical centers. Casgevy will cost $2.2 million.

Beta thalassemia is a hereditary disease that is characterized by a genetic defect in the synthesis of beta polypeptide chains, which leads to impaired synthesis of hemoglobin A. This is fraught with a number of consequences – from the development of severe anemia to bone marrow hyperactivity. Treatment today includes lifelong red blood cell transfusions, removal of the spleen, or the use of iron chelators.

In 2022, the FDA approved Bluebird Bio’s gene therapy Zynteglo for beta thalassemia. The administration of this drug differs from Casgevy: stem cells from the patient’s bone marrow are treated with viral vectors containing the desired hemoglobin gene and returned back to the body. Zynteglo price: $2.8 million.

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