Trial Tests a New Gene-Editing Strategy for Sickle Cell Disease

The multicenter trial is evaluating a CRISPR-Cas9 approach designed to directly modify the beta‑globin gene in sickle cell disease.

FDA-approved gene therapies have shown that modifying a patient’s own blood‑forming stem cells can significantly reduce complications of sickle cell disease. Now, an early‑stage clinical trial is studying a different approach—one designed to directly modify the gene that causes the disease.