CRISPR Therapeutics and Vertex Pharmaceuticals Deliever New CTX001 Data
Gene therapy took another public step forward during a virtual presentation at a scientific conference on Friday. CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals (NASDAQ: VRTX) presented updated results from studies with CTX001, the first CRISPR-based gene-editing therapy to reach human-stage testing.
In the Climb-111 trial treating transfusion-dependent beta-thalassemia (TDT), CTX001 drastically reduced patients' dependence on frequent blood transfusions. In the years leading up to the trial, Patient 1 required 34 units of red blood cells annually and Patient 2 needed transfusions at a rate of 61 units per year. At 15 months following a single infusion of CTX001, patient 1 still hasn't required a transfusion. At the five-month mark, patient 2 hasn't required a transfusion either.
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