Vertex Pharmaceuticals Incorporated: Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress
Both trials met the primary and key secondary endpoints at the pre-specified interim analysis - Data continue to demonstrate transformative and durable benefit - Safety profile consistent with busulfan