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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]
Sickle cell disease, often called sickle cell anemia, which is its most serious form, is caused by a genetic disorder that alters the ability of red blood cells to deliver oxygen to every part of ...
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.
[92] [93] The risks and benefits related to gene therapy for sickle cell disease are not known. [93] Gene therapy has been used in the eye. The eye is especially suitable for adeno-associated virus vectors. Voretigene neparvovec is an approved gene therapy to treat Leber's hereditary optic neuropathy.
By Leroy Leo and Bhanvi Satija (Reuters) -The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the ...
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 .