Data Presentation on transfusion-dependent β-thalassemia or severe sickle cell disease at the 2022 European Hematology Association Congress from Vertex and CRISPR Therapeutics

Vertex Pharmaceuticals Inc. (VRTX) and CRISPR Therapeutics (CRSP) presented new data on exa-cel (CTX001™) at the European Hematology Association (EHA) Congress. Forty-four patients with transfusion-dependent β-thalassemia (TDT) and thirty-one with severe sickle cell disease (SCD) were monitored from 1.2 to 37.2 months after exa-cel dosing. TDT patients had substantial mean increases in fetal hemoglobin (HbF) and corresponding increases in mean total hemoglobin (Hb), while all severe SCD were free of VOCs and mean HbF of approximately 40% by month 3 and 4 respectively. The data from 75 patients demonstrate that exa-cel has the potential to be a one-time functional cure, with safety profile be consistent with myeloablative conditioning and autologous stem cell transplant. For more information, press release.