An investigational gene therapy for Sickle Cell Disease (SCD) and β-thalassaemia, known as ARU-1801, has been given Orphan Drug Designation an orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development.
The therapy is expected to increase functioning red blood cells through proprietary technology that inserts a modified fetal haemoglobin gene into autologous stem cells via a lentiviral vector. A phase 1/2 clinical study in 10 individuals with sickle cell disease is currently examining the efficacy and safety of ARU-1801.
„We are excited to build on the momentum afforded by both today’s announcement of Orphan Drug status as well as our recent announcement of Rare Pediatric Disease status for ARU-1801,“ said Will Chou, M.D., Chief Executive Officer of Aruvant. „For patients suffering from sickle cell disease, we believe the ultimate promise of gene therapy is a one-time cure without the side effect profile of high intensity myeloablative conditioning. We are committed to providing patients with that option and look forward to presenting more data on our Reduced Intensity Conditioning (RIC) approach as patients continue to be treated in our ongoing Phase 1/2 study.“