Nearly a year after its approval, the first medical treatment using Nobel Prize-winning Crispr technology is now being administered to patients.
Called Casgevy, the gene-editing treatment is intended for people with sickle cell anemia and a related blood disorder called beta thalassemia. Regulators in the United Kingdom approved the treatment in November 2023, followed by those in the United States and Europe in December. Vertex, the pharmaceutical company that markets Casgevy, announced in a Nov. 5 earnings call that the first person to receive Casgevy outside of a clinical trial was dosed in the third quarter of this year. The company reported $2 million in revenue from that patient. (Casgevy debuted with a price tag of $2.2 million in the United States.)
“Cagevy has been enthusiastically received by patients, clinicians and policymakers, and the launch is gaining momentum in all regions,” said Stuart Arbuckle, Vertex’s chief operating officer, on the earnings conference call. He added that 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 have undergone cell collections before receiving treatment, up from 20 patients last quarter.
In sickle cell anemia 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 anemia have hard, crescent-shaped red blood cells that stick together and block blood flow, causing extreme attacks of pain. These pain crises can last hours or days and can land patients in the hospital. In beta thalassemia, the body does not produce enough hemoglobin, causing anemia. People with beta thalassemia severe need regular blood transfusions every few weeks throughout their lives.
Casgevy works by using Crispr to modify a person’s own cells so that they produce a healthy type of hemoglobin.
The delay in patients receiving Casgevy is not necessarily unexpected, as the treatment is complex to administer and only certain hospitals can perform the procedure. On last week’s earnings conference call, Arbuckle said there are now 45 treatment centers authorized to administer Casgevy, and Vertex expects that number to increase to about 75 worldwide.