Learn how gene editing is transforming sickle cell disease treatment and why expanding access to younger children matters.
Regulators have opened a one-time gene therapy to some of the youngest patients with sickle cell disease. On July 1, 2026, ...
Casgevy is now approved in the U.S. for children with sickle cell as young as 2, becoming the first gene-editing therapy for ...
Vertex Pharmaceuticals and CRISPR Therapeutics' gene-editing drug is the only sickle cell disease gene therapy on the market for young children.