Gene Therapy Trials Show Positive Results in Sickle Cell Disease and Thalassemia
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JANUARY 30, 2024
Results from the phase 1/2 RUBY and EdiThal trials suggest that CRISPR/CASP 12 gene editing has the potential to cure sickle cell disease and transfusion-dependent beta-thalassemia (TDT). The findings, presented in a poster at the 2023 American Society of Hematology conference, indicate the safety and efficacy of infusion of renizgamglogene autogedtemcel (EDIT-301), now called “Reni-Cel,” in 11 patients with severe sickle cell disease and six patients with TDT.
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