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DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. 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.
The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its ...
CRISPR gene editing (CRISPR, ... In December 2023, the FDA approved the first gene therapy in the US to treat patients with Sickle Cell Disease (SCD). The FDA ...
The FDA approves the first of two gene therapies this month to treat sickle cell disease. ... which is the first to use the CRISPR gene editing technology that won a Nobel prize in 2020 ...
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. [ 1 ][ 2 ][ 3 ] The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans ...
The FDA is expected to issue a final ruling by Dec. 8. No drug that uses CRISPR gene-editing — which was invented in 2009 — has been granted FDA approval. What’s more, Tuesday’s meeting ...
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