ASH 2020: CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope
Published: Dec. 7, 2020
BioSpace
CRISPR Therapeutics and Vertex Pharmaceuticals presented new data on 10 patients treated with CTX001, which demonstrated a consistent and sustained response, in transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). CTX001 is an investigational CRISPR/Cas9-based gene-editing therapy.
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