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  2. FDA approves cure for sickle cell disease, the first ... - AOL

    www.aol.com/news/fda-approves-cure-sickle-cell...

    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 ...

  3. FDA considers first CRISPR gene editing treatment that may ...

    www.aol.com/fda-considers-first-crispr-gene...

    With sickle cell disease — also called sickle cell anemia — red blood cells take on a folded or sickle shape that can clog tiny blood vessels and cause progressive organ damage and pain, and ...

  4. FDA moves closer to sickle cell cure that uses gene editing - AOL

    www.aol.com/news/fda-moves-closer-sickle-cell...

    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 ...

  5. CRISPR gene editing - Wikipedia

    en.wikipedia.org/wiki/CRISPR_gene_editing

    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]

  6. Exagamglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Exagamglogene_autotemcel

    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.

  7. Sickle cell disease - Wikipedia

    en.wikipedia.org/wiki/Sickle_cell_disease

    Sickle cell disease (SCD), also simply called sickle cell, is a group of hemoglobin-related blood disorders typically inherited. [2] The most common type is known as sickle cell anemia. [2] It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2] This leads to a rigid, sickle -like shape under ...

  8. FDA approves two gene therapies for sickle cell, bringing ...

    www.aol.com/news/fda-expected-approve-first...

    The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...

  9. US FDA approves two gene therapies for sickle cell disease - AOL

    www.aol.com/news/us-approves-two-gene-therapies...

    (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 ...

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