Sickle Cell Disease Gene Therapy Receives Orphan Drug Designation

ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD) and beta thalassemia, was granted orphan drug designation by the FDA.

ARU-1801 is designed to increase the number of a patient’s functional red blood cells by inserting a modified version of the fetal hemoglobin gene directly into the patient’s own stem cells using a lentiviral vector. Its application is supported by data from an ongoing, open-label, phase I/II trial evaluating ARU-1801’s safety and efficacy in children and adults with SCD.

Source: Aruvant Sciences press release, January 22, 2020.