Casgevy (exagamglogene autotemcel), the pioneering gene editing therapy based on CRISPR technology, has received approval from the US Food and Drug Administration (FDA) for the treatment of patients aged 12 and above with transfusion-dependent β-Thalassemia (TDT). This marks a significant milestone as Casgevy becomes the world’s first CRISPR-based gene editing therapy to gain FDA approval.
Co-developed by Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP), Casgevy is an autologous cell therapy indicated for the treatment of sickle cell disease (SCD) and TDT. The therapy carries a price tag of USD 2.2 million, aligning with its status as a groundbreaking treatment. Casgevy was previously approved in the US in December last year for the treatment of SCD patients aged 12 and above with recurrent vascular occlusion crises.- Flcube.com
