Vertex Reports Durable Results for Casgevy in Sickle Cell and Thalassemia

Vertex Pharmaceuticals’ gene therapy Casgevy (exagamglogene autotemcel) has shown sustained benefits in patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) across three Phase III trials.

All 45 SCD patients achieved at least 12 consecutive months without hospital visits, averaging 36.1 months, and 95.6% remained free from vaso-occlusive crises for a year. Among 55 TDT patients, 98.2% achieved transfusion independence for at least 12 months, with a mean haemoglobin of ≥9g/dL.

The treatment’s safety profile remains consistent with conditioning using busulfan and stem cell transplant.

Dr. Kevin Kuo (University of Toronto) praised the long-term results as a milestone for patients with limited treatment options.

Casgevy, the first approved CRISPR/Cas9 therapy, is authorised in several countries, including the U.S., UK, and UAE, though Canada is still reviewing it due to cost concerns.

10-07-2025