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The FDA approved a new treatment for sickle cell disease. The therapy is first to use the ground-editing tool CRISPR.
Sickle cell is the most common disease to receive approval for gene therapy treatment, following decades of development and years of approvals for therapy for people with rarer conditions.
Voxelotor. Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. [1][3][4][5][6] Voxelotor is the first hemoglobin oxygen-affinity modulator. [7] Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell ...
Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [8]
(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 United States based on the Nobel ...
UNII. 2C6A9NH2Z8. KEGG. D12765. Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1][3][4][5] The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells ...
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