Vertex presents new data on Casgevy at ASH

US biopharma Vertex Pharmaceuticals (Nasdaq: VRTX) today announced data from multiple studies demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in people ages five years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).

The results, including the first presentation of clinical data from pivotal studies in children ages five-11 years, and longer-term data from the pivotal studies of people with severe SCD and TDT ages 12 years and older, are being presented at the American Society of Hematology (ASH) annual meeting. Casgevy is currently approved for eligible people ages 12 years and older with SCD or TDT in the USA, UK, the European Union, Saudi Arabia, Bahrain, Kuwait, Qatar, Canada, Switzerland and the United Arab Emirates.

Gene therapy has rapidly evolved, and Casgevy is considered a potential blockbuster by its developers. Still, real-world uptake remains slow, largely due to the conditioning process needed before infusion. Casgevy third-quarter 2025 sales came up way short at $17 million, where Wall Street had predicted $43 million.