Almost a year after its approval, The first medical treatment that uses CRISPR gene editing technology is being administered to patientsawarded the Nobel Prize.
What is Casgevy
Named Casgevygene editing treatment is intended for people with sickle cell anemia and a blood disorder related called beta thalassemia. British regulators approved the treatment in November 2023, followed by US and European regulators in December. Vertex, the pharmaceutical company that markets Casgevy, announced in an earnings call on Nov. 5 that the first person to receive Casgevy outside of a clinical trial got the dose in the third quarter of this year. The company reported revenue of $2 million from that patient. Casgevy debuted with a price of $2.2 million in the US.
“Casgevy has been enthusiastically received by patients, clinicians and policymakers, and the launch is gaining momentum in all regions,” said Stuart Arbuckle, COO of Vertex, on the earnings call. He added that more patients are accessing the treatment commercially.
When WIRED contacted Vertex by email, spokesperson Eleanor Celeste declined to provide the exact number of patients who have received Casgevy. However, the company claims that 40 patients have undergone cell extractions before receiving the treatmentcompared to 20 last quarter.
Sickle cell anemia and Beta thalassemia
In sickle cell anemia and beta thalassemia, patients do not produce healthy hemoglobinthe substance in red blood cells responsible for transporting oxygen throughout the body. The cause is errors in the hemoglobin gene. As a result, people with sickle cell anemia have ‘hard’, crescent-shaped red blood cells that stick together and block blood flow, causing extreme pain attacks. Are pain crisis They can last hours or days and can put 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, since administering the treatment is complex and only a few hospitals can perform the procedure. In last week’s earnings call, Arbuckle said that 45 treatment centers are now authorized to administer Casgevyand Vertex expects that number to increase to approximately 75 worldwide.
How does the treatment work?
The treatment is carried out by extracting the patient’s own ‘hematopoietic’ stem cells and sending them to a laboratory for editing. Before receiving an infusion of the cells, patients must undergo chemotherapy to prepare their bone marrow for the new cells. Once infused, the edited cells travel to the marrow, where they start to produce new red blood cells with healthy hemoglobin.
In clinical trials, Casgevy greatly reduced or eliminated debilitating pain attacks in people with sickle cell anemia, and enabled most beta thalassemia patients to stop blood transfusions. For some, that amounts to a functional cure.
Vertex estimates that about 35,000 patients with sickle cell anemia and beta thalassemia in the US and Europe could benefit from Casgevy. The company is investing in additional manufacturing capacity for the treatment, and in September it gained approval for a third manufacturing facility, Arbuckle said.
A major obstacle for those interested in receiving Casgevy remains the need to undergo chemotherapywhich can cause infertility. Another obstacle is the long and arduous process of collecting cells and staying in the hospital for weeks after receiving the edited cells. The first CRISPR drug is herebut it remains to be seen how popular it will be with patients.
Article originally published in WIRED. Adapted by Mauricio Serfatty Godoy.
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