FDA approves Casgevy for transfusion-dependent beta thalassemia

CRISPR Therapeutics/Casgevy (exa-cel)

CRISPR Therapeutics announced FDA approval of Casgevy for transfusion-dependent beta thalassemia on 16 January 2024, weeks after sickle cell approval. The one-time treatment edits blood stem cells to free patients from regular transfusions.

• Casgevy is the first CRISPR-based therapy approved; it edits BCL11A or KLKB1 genes to restore fetal hemoglobin.
• Approved for patients ≥12 years; ~1,000 patients in US age 12+ eligible for treatment.
• Transfusion-dependent patients require monthly or more frequent blood transfusions; Casgevy provides permanent correction.
• Approval followed by sickle cell disease (December 2023) and precedes EU/UK/Bahrain approvals in following months.