Pipeline

Pipeline

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ARU-1801

ARU-1801 is an investigational gene therapy for sickle cell disease and β-thalassemia. ARU-1801 utilizes proprietary technology intended to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vector. Studies have indicated that sickle cell patients with elevated levels of fetal hemoglobin have fewer vaso-occlusive crises and hospitalizations, due in part to improved characteristics of fetal hemoglobin relative to adult hemoglobin. ARU-1801 is the only known clinical-stage gene therapy to deliver the gene encoding fetal hemoglobin, which has been modified to optimize oxygen carrying capacity and anti-sickling properties.