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

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that both pivotal trials for exagamglogene autotemcel in patients with transfusion-dependent beta thalassemia or severe sickle cell disease met primary and key secondary endpoints at pre-specified interim analyses.