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.
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