FDA approves Casgevy for sickle cell disease in patients 12 and older

CRISPR Therapeutics/Casgevy (exa-cel)

The FDA approved Casgevy and bluebird bio's Lyfgenia, the first cell-based gene therapies for sickle cell disease, for patients 12 and older. Casgevy became the first FDA-approved treatment using CRISPR genome editing.

• Casgevy edits blood stem cells to produce fetal hemoglobin, eliminating sickle vaso-occlusive crises.
• In clinical studies, 29 of 31 evaluated patients (93.5%) achieved freedom from further pain crises.
• One-time, cell-based therapy contrasts with lifelong symptomatic or blood transfusion management.
• Historic approval: first CRISPR therapy approved in US, opening era of gene-edited cell therapeutics.