Transformative Benefits Prove Durable in Sickle-Cell Diseaseand Transfusion-Dependent Beta Thalassemia; Reiterate BUY Rating

Vertex provided a program update on CASGEVYTM, the first and to date only approved CRISPR/Cas9 gene-edited therapy, at the American Society of Hematology (ASH) Annual Meeting this week, revealing data from long-term follow-up of patients who participated in clinical trials.

Transformative benefits of CASGEVY proved durable in both sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).• CASGEVY’s safety profile is consistent with busulfan conditioning and autologous hematopoietic stem cell transplant.

Efforts to secure regulatory clearance in additional countries, improve patient access, and broaden reimbursement coverage are ongoing.