Casgevy (exa-cel)

Casgevy (exa-cel) — sickle cell & beta-thalassemia. Genetic & blood disorders. Reviewed Jul 2026.

Ended pain crises or transfusion dependence for most patients in uncontrolled trials, at very high cost and with intensive conditioning.

Stage

Approved

Rung: Available now. Approved or established. Usable today, with a clinician.

Evidence grade

uncontrolled, large effect

Small or uncontrolled human data, or strong animal data. How grades work.

What it is

A CRISPR/Cas9 therapy that edits a patient's own blood stem cells to switch fetal hemoglobin back on, ending the disease.

The evidence

Clinical trials: eliminated recurrent vaso-occlusive pain crises in sickle cell and transfusion dependence in beta-thalassemia for the large majority of patients. Requires intensive conditioning; very high cost and access limits.

What you can do today

This is available today. Whether it suits you depends on your health, your other medicines and your risks, so discuss it with a clinician who knows your history before starting or changing anything.

Sources

Official or company statementfda.gov

No primary paper or registry record is linked for this entry yet. The evidence summary names the studies it relies on.

Change history

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