NHS Approves Groundbreaking Gene-Editing Therapy for Beta Thalassemia

Casgevy, the first CRISPR-based treatment, offers potential cure for hundreds of patients reliant on regular blood transfusions.

The National Institute for Health and Care Excellence (NICE) has approved Casgevy for NHS use in England.
Casgevy employs CRISPR technology to modify faulty genes in bone marrow stem cells.
The treatment is aimed at patients with transfusion-dependent beta thalassemia (TDT), potentially eliminating the need for regular transfusions.
The therapy will be available at a discounted rate through a commercial agreement between the NHS and Vertex.
Up to 460 eligible patients will receive the treatment initially, with data collection on its effectiveness ongoing.