About a year after its approval First medical treatment that uses Nobel Prize-winning technology crisper Now it is being given to patients.
The gene-editing treatment, called CasGevi, is for people with sickle cell disease and a related blood disorder called beta thalassemia. UK regulator Treatment approved in November 2023, Next is America And Europe in December. Vertex, the pharmaceutical company that markets Casgevi, announced in a Nov. 5 earnings call that the first person to receive Casgevi outside of clinical trials was dosed in the third quarter of this year. The company reported $2 million in revenue from that patient. (The Kasegavi debuted in the US with a price tag of $2.2 million.)
“Kezvi has been enthusiastically received by patients, physicians and policymakers, and the launch is gaining momentum across all regions,” Stuart Arbuckle, Vertex's chief operating officer, said on the earnings call. Additional patients are using the treatment commercially, he said.
When WIRED contacted Vertex via email, spokeswoman Eleanor Celeste declined to provide the exact number of patients receiving Casgevi. However, the company says 40 patients have undergone cell collection in anticipation of receiving the treatment, up from 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 responsible. As a result, people with sickle cell have hard, crescent-shaped red blood cells that stick together and block blood flow, causing extreme pain. These pain crises can last for hours or days and can send patients to the hospital. In beta thalassemia, the body does not make enough hemoglobin, leading to anemia. People with severe beta thalassemia require regular blood transfusions every several weeks throughout their lifetime.
CasGevi works by using CRISPR to modify a person's own cells so they can produce a healthy type of hemoglobin.
Delays in patients receiving CasGevi are not necessarily unexpected, as the treatment is complex and only a few hospitals are able to perform the procedure. On last week's earnings call, Arbuckle said 45 treatment centers are now authorized to administer Casgevi, and Vertex expects that number to grow to about 75 worldwide.