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The first Crispr treatment arrives in patients
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The first Crispr treatment arrives in patients

Almost a year after its approval, the first medical treatment using Nobel Prize-winning technology Crisp is now distributed to patients.

Called Casgevy, the gene-editing treatment is for people with sickle cell disease and a related blood disorder called beta thalassemia. British regulators approved treatment in November 2023, followed by the United States and Europe in December. Vertex, the pharmaceutical company that markets Casgevy, announced in a conference call on November 5 that the first person to receive Casgevy outside of a clinical trial received a dose in the third quarter of this year. The company reported $2 million in revenue from this patient. (Casgevy debuted with a $2.2 million price tag in the United States.)

“Cagevy has been enthusiastically received by patients, physicians and policymakers, and the launch is gaining momentum across all regions,” Vertex COO Stuart Arbuckle said on the call. to the results. He added that more and more patients are accessing the treatment commercially.

When WIRED followed up with Vertex via email, spokesperson Eleanor Celeste declined to provide the exact number of patients who received Casgevy. However, the company says 40 patients underwent cell harvesting in anticipation of receiving the treatment, compared to 20 patients last quarter.

In sickle cell disease and beta thalassemia, patients do not produce healthy hemoglobin, the substance in red blood cells responsible for carrying oxygen throughout the body. Errors in the hemoglobin gene are to blame. As a result, people with sickle cell disease have hard, crescent-shaped red blood cells that stick together and block blood flow, causing extreme bouts of pain. These painful attacks can last hours or days and land patients in the hospital. In beta thalassemia, the body does not produce enough hemoglobin, leading to anemia. People with severe beta thalassemia need regular blood transfusions every several weeks throughout their lives.

Casgevy uses Crispr to modify a person’s own cells so that they produce a healthy type of hemoglobin.

The delay in the arrival of patients receiving Casgevy is not necessarily unexpected, as the treatment is complex to administer and only certain hospitals are able to perform the procedure. On last week’s earnings call, Arbuckle said 45 treatment centers are now authorized to administer Casgevy, and Vertex expects that number to rise to about 75 worldwide.