First CRISPR Gene Therapy Cures Sickle Cell Disease Patients

In a historic milestone for medicine, patients with sickle cell disease are being cured through CRISPR gene editing therapy. Casgevy, approved by the FDA in late 2023, has now treated hundreds of patients with remarkable success.

Victoria Gray, the first American to receive the treatment, recently celebrated five years without a sickle cell crisis — something unimaginable before. "I feel like a new person," she said. "I can do things I never could before."

“Casgevy, approved by the FDA in late 2023, has now treated hundreds of patients with remarkable success.”

The therapy works by editing patients' own stem cells to produce fetal hemoglobin, which prevents the sickling of red blood cells. While expensive, the one-time treatment offers a permanent cure for a disease that affects millions worldwide.