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December 8, 2023 at 1:19 PM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the ...
About 20,000 people in the US are thought to have a severe enough form of the disease to potentially qualify for a treatment like this. “To have sickle cell suddenly be the focus of this ...
The drug, called exa-cel, treats sickle cell disease, an inherited blood disorder that affects an estimated 100,000 people in the U.S., most of whom are Black, according to the Centers for Disease ...
Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1] [3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. [7]
The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease. The groundbreaking ...
In 2023, the first drug making use of CRISPR gene editing, Casgevy, was approved for use in the United Kingdom to cure sickle-cell disease and beta thalassemia. [13] [14] Casgevy was approved for use in the United States on December 8, 2023, by the Food and Drug Administration. [15]
Victoria Gray. Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1] This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia. [1]
The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics.
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