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The original was posted on /r/singularity by /u/IluvBsissa on 2024-01-17 01:27:40+00:00.
"Transfusion-dependent beta thalassemia is a serious genetic disorder that hinders the production of hemoglobin in the blood and requires regular blood transfusions for treatment.
Casgevy uses the novel CRISPR gene-editing technology to modify patients’ blood cells and transplant the modified cells back into the bone marrow, triggering an increase in the production of hemoglobin, according to the FDA.
The most common side effects were mouth sores, fever caused by a low level of white blood cells and decreased appetite, the FDA said.
In a statement, Vertex CEO and president Reshma Kewalramani said the company looks forward “to bringing Casgevy to eligible patients who are waiting.”
Vertex says it’s engaging with experienced hospitals to establish “a network of independently operated, authorized treatment centers” throughout the U.S. to administer Casgevy. There are currently nine activated treatment centers in the U.S., but more will be activated in the coming weeks, Vertex said in a press release. The administration of Casgevy requires experience in stem cell transplantation."