$3 million prize goes to duo whose research led to first sickle cell CRISPR therapy.
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Posted Apr. 20, 2026, 12:16 AM

The biotech company Vertex then used the cut-and-paste gene-editing tool CRISPR to snip out the repressor region of BCL11A. This work eventually led to the development of Casgevy. Administering the therapy involves extracting a person's bone marrow cells, editing the BCL11A repressor using CRISPR, and then reinfusing the gene-edited bone marrow cells back into the patient. The edited cells begin to make red blood cells with high levels of fetal hemoglobin..