CRISPR Therapeutics reports FDA approval of Casgevy from age two

CRISPR Therapeutics stated in its second-quarter business update, issued on 3 August 2026, that the US Food and Drug Administration has approved Casgevy (exagamglogene autotemcel) for children two years of age and older with sickle cell disease or transfusion-dependent beta thalassemia. The company describes it as the first genetic therapy indicated for children as young as two for both conditions, says the decision came 53 days after filing, and puts the number of patients newly eligible in the United States at approximately 5,500.

Casgevy is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy. A patient’s own haematopoietic stem and progenitor cells are edited at the erythroid-specific enhancer region of the BCL11A gene, which raises production of fetal haemoglobin in red blood cells. Before this decision it was approved for patients aged 12 and older, in 39 countries across North America, Europe and the Middle East.

The company reported $76 million in Casgevy revenue for the quarter, up 78 per cent on the previous quarter and 151 per cent year on year. It also said it has begun Phase 1 trials of CTX340, targeting angiotensinogen in refractory hypertension, and of CTX460, targeting SERPINA1 in alpha-1 antitrypsin deficiency and the first candidate from its SyNTase editing platform.

The approval date and the review-time figure come from the company’s own release; no corresponding FDA announcement was located for this capsule.