One-time gene therapy for sickle cell disease

Aruvant is developing ARU-1801, a one-time, potentially curative gene therapy for sickle cell disease and β-thalassemia. In an ongoing clinical phase 1/2 study, ARU-1801, administered with only reduced intensity conditioning, has provided stable reductions in disease burden and opioid dependence.

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Aruvant Sciences

NA, Aruvant

Aruvant Sciences is a clinical-stage gene therapy company focused on hematological conditions, with an emphasis on helping patients suffering from sickle cell disease and β-thalassemia. ARU-1801, the lead candidate in Aruvant's pipeline, is an investigational lentiviral gene therapy for sickle cell disease and transfusion-dependent β-thalassemia. ARU-1801 incorporates a patented gene payload for a modified gamma-globin delivered into autologous stem cells via a proprietary vector construct, with the aim of restoring normal red blood cell function through increased levels of fetal hemoglobin. The high potency of the modified gamma globin enables ARU-1801 engraftment with only Reduced Intensity Conditioning (RIC).

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