Advanced Therapies Week 2022 | ARU-1801, a novel lentiviral gene therapy for SCD

Current investigational therapies for sickle cell disease (SCD) require conditioning chemotherapy such as busulfan to administer the genetically-modified stem cells in the patient’s bone marrow. Will Chou, MD, Aruvant, New York, NY, provides an overview of ARU-1801, for patients with SCD. The lentiviral gene therapy uses fewer doses of an alternative chemotherapy, melphalan, which results in fewer side effects such as thrombocytopenia and veno-occlusive liver disease, as well as shorter hospital stays. This interview took place at Advanced Therapies Week 2022.