Updated results announced Saturday from two clinical trials involving an experimental, genome-editing treatment showed prolonged benefit for patients born with inherited blood disorders sickle cell disease and beta-thalassemia.
Seventy-five patients have now been treated with the therapy developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics that uses CRISPR technology to genetically fix diseased cells. Following a one-time infusion, all 31 of the patients with sickle cell reported being free from episodes of severe pain; 42 of the 44 patients with beta-thalassemia no longer required blood transfusions.
In both studies, the positive outcomes are essentially functional cures of the respective inherited diseases, although just under half of the 75 patients have been followed for at least one year — the minimum time needed to confirm the benefit.
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