The Food and Drug Administration has granted Orphan Drug designation to ARU-1801 (Aruvant) for the treatment of sickle cell disease.

The investigational gene therapy is expected to increase functioning red blood cells through proprietary technology that inserts a modified fetal hemoglobin 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.

“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,” said Will Chou, MD, Chief Executive Officer of Aruvant.

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Orphan Drug status is granted to new therapies that treat diseases impacting ≤200,000 individuals in the US. 

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