almost 1 year Following its approval, the first medical treatments using the Nobel Prize-winning technology Crispr are now being delivered to patients.
The gene-editing treatment, called Kasgeby, is aimed at people with sickle cell disease and a related blood disorder called beta-thalassemia. British regulators approved the treatment in November 2023, followed by the US and Europe in December. Vertex, the pharmaceutical company that sells Casgevy, announced in an earnings call on November 5 that the first person to receive Casgevy outside of a clinical trial was administered in the third quarter of this year. The company reported $2 million in revenue from its patients. (Kasgeby debuted in the U.S. with a $2.2 million price tag.)
“Cagevy has been enthusiastically received by patients, physicians and policymakers, and the launch is gaining momentum in all regions,” Stuart Arbuckle, Vertex’s chief operating officer, said on the earnings call. . He added that more patients are accessing this treatment commercially.
When WIRED followed up with Vertex via email, spokeswoman Eleanor Celeste declined to reveal the exact number of patients treated with Casgeby. However, the company said 40 patients had their cells harvested in hopes of receiving treatment, up from 20 in the previous quarter.
In sickle cell disease and beta-thalassemia, patients do not produce healthy hemoglobin, a substance in red blood cells that is responsible for transporting oxygen throughout the body. It is caused by an error in the hemoglobin gene. As a result, sickle cell patients develop hard, crescent-shaped red blood cells that stick together and block blood flow, causing severe pain. These pain crises can last for hours or days and can result in patients being hospitalized. In beta-thalassemia, the body does not produce enough hemoglobin, causing anemia. Patients with severe beta-thalassemia require regular blood transfusions every few weeks for the rest of their lives.
Casgevy uses Crispr to modify an individual’s cells so that they produce a healthy type of hemoglobin.
Delays in patients receiving Kasgeby treatment are not necessarily unexpected, as Kasgeby treatment is complex to administer and can only be performed by certain hospitals. Arbuckle said on an earnings call last week that 45 treatment centers are currently authorized to administer Kasugevy, and Vertex expects that number to grow to about 75 worldwide.
