🧬 First CRISPR treatment is now being given to patients

• The first treatment using Nobel Prize-winning CRISPR technology has begun being administered to patients with sickle cell disease and beta-thalassemia.
• Patients do not produce healthy hemoglobin, the substance in red blood cells that transports oxygen in the body.
• Casgevy uses CRISPR to modify the patient's own cells so they produce healthy hemoglobin.

Treatment now reaching patients worldwide

The Nobel Prize-winning CRISPR technology is now being used in the first approved medical treatment, Casgevy. The treatment is aimed at people with sickle cell disease and beta-thalassemia, two serious blood disorders, reports Wired.

After approvals in the UK, US, and Europe at the end of 2023, the first patient outside clinical trials received the treatment during the third quarter of 2024. Vertex, the pharmaceutical company behind Casgevy, reported revenue of 2 million dollars from this patient.

How the treatment works

In sickle cell disease and beta-thalassemia, patients do not produce healthy hemoglobin, the substance in red blood cells that transports oxygen in the body. People with sickle cell disease develop hard, crescent-shaped red blood cells that clump together and block blood flow, causing severe pain attacks. In beta-thalassemia, the body doesn't produce enough hemoglobin, leading to anemia and the need for regular blood transfusions.

Casgevy uses CRISPR to modify the patient's own cells so they produce healthy hemoglobin. The treatment is carried out by collecting the patient's blood-forming stem cells and sending them to a laboratory for editing. Patients then undergo cell therapy to prepare their bone marrow for the new cells.

Upscaling in progress

45 treatment centers are now authorized to administer Casgevy, and Vertex expects this number to increase to approximately 75 worldwide. The company estimates that around 35,000 patients with sickle cell disease and beta-thalassemia in the US and Europe could benefit from the treatment.

In September, Vertex received approval for a third manufacturing facility to meet demand.

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