Vertex Pharmaceuticals announced the presentation of longer-term data for CASGEVY (exagamglogene autotemcel) from global clinical trials at the American Society of Hematology (ASH) Annual Meeting and Exposition. These data further demonstrate the transformative and durable clinical benefits of the CRISPR/Cas9 gene-edited therapy.
The longest follow-up for both sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) patients now extends beyond 5 years, with median follow-up times of 33.2 months and 38.1 months, respectively. The safety profile observed was consistent with busulfan conditioning and autologous hematopoietic stem cell transplant.
Vertex reported that more than 45 authorized treatment centers have been activated globally to support CASGEVY delivery, and over 40 patients have initiated their first cell collection. The company is actively working with reimbursement authorities to secure sustainable access for patients worldwide.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.