Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would advance one day.

Updated Data from 17 Patients Consistent with Previously Presented Data; All Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of ...

Victoria Gray, the first person cured of sickle cell using CRISPR gene editing therapies, spoke in Fargo about her journey and the need to lower the $2M treatment cost.

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...