Vertex/CRISPR Partnered Cell Therapy Shows Encouraging Response In Sickle Cell Disease, Thalassemia Patie
Benzinga Vertex Pharmaceuticals IncorporatedVRTX andCRISPR Therapeutics AGCRSP announced that pivotal trials for exagamglogene autotemcel (exa-cel) for transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD) met primary and key secondary endpointsat pre-specified interim analyses.
Of the 48 patients with TDT who had received exa-cel at the time of the analysis, 24/27 (88.9%) achieved the primary endpoint of transfusion independence for at least 12 consecutive months (TI12) and the secondary endpoint of transfusion independence for at least 6 consecutive months (TI6) with mean weighted hemoglobin of at least 9 g/dL.
The mean duration of transfusion independence was 20.5 months, with a maximum of 40.7 months.
The mean proportion of editedalleles was stable in the bone marrow and peripheral blood over time, indicating successful permanent editing in long-term hematopoietic stem cells.
Of the 35 patients with SCD who had received exa-cel at the time of the analysis, 16/17 (94.1%) achieved the primary endpoint of freedom from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12).
