New FDA nod for Vertex and CRISPR’s Casgevy
Just a month after winning its first US approval for sickle cell disease (SCD), Casgevy (exagamglogene autotemcel [exa-cel]) has won a second Food and Drug Administration (FDA) approval.
This time, the CRISPR/Cas9 gene-edited cell therapy from Vertex Pharmaceuticals (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP), has been approved for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
The approval opens up the one-time treatment to around 1,000 American patients with the serious, life-threatening genetic disease.
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