In a historic milestone for genetic medicine, the FDA has approved Casgevy—a CRISPR-based gene-editing therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics—for the treatment of sickle cell disease. The approval marks the first time a CRISPR therapy has been authorized in the United States, opening new possibilities for patients with inherited blood disorders.

The U.S. Food and Drug Administration has granted approval to Casgevy (exagamglogene autotemcel), a groundbreaking CRISPR-based gene-editing therapy for sickle cell disease, jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This landmark decision, announced on October 9, 2024, makes Casgevy the first CRISPR therapy to receive FDA approval in the United States.

Casgevy is designed to edit the BCL11A gene in patients’ hematopoietic stem cells, enabling the production of fetal hemoglobin and thereby reducing the painful vaso-occlusive crises that characterize sickle cell disease. The therapy is administered as a one-time treatment, and clinical trial data show that the majority of patients experienced freedom from severe pain crises for at least 12 months post-treatment.

“This approval is a historic step for the field of gene editing and for patients with sickle cell disease,” said Dr. Reshma Kewalramani, CEO and President of Vertex Pharmaceuticals. “Casgevy offers the possibility of a functional cure for a disease that has seen little therapeutic innovation in decades.”

Dr. Samarth Kulkarni, CEO of CRISPR Therapeutics, added, “This moment validates years of scientific discovery and collaboration. We are grateful to the patients and families who participated in the clinical trials and made this achievement possible.”

The approval follows a series of positive recommendations from FDA advisory committees and builds on prior regulatory authorizations in the UK and EU. Casgevy’s approval is expected to pave the way for further CRISPR-based therapies targeting other genetic disorders.