05:13 AM EDT, 06/14/2024 (MT Newswires) -- Vertex Pharmaceuticals ( VRTX ) said Friday its Casgevy gene-editing therapy demonstrated clinical benefits in trials in sickle cell disease and transfusion-dependent beta thalassemia.

In sickle cell disease, about 92% of evaluable patients were shown to be free from vaso-occlusive crises, or VOCs, for at least 12 months, while 97% of patients with 16 months of follow-up were free from hospitalizations related to VOCs for at least 12 months, the company said.

In transfusion-dependent beta thalassemia, 94% of evaluable patients were shown to be transfusion-independent for at least 12 months, while all patients dosed with at least 16 months of follow up were transfusion free, the company said.

Patients in both studies reported sustained and "clinically meaningful" improvements in quality of life and overall health status, Vertex said.

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