Aruvant’s lead candidate, ARU-1801, consists of autologous cells that are genetically modified with a lentiviral vector that encodes a novel, highly potent anti-sickling γ-globin. ARU-1801 was ...

To improve the efficacy of gene therapy when using anti-sickling beta globin gene transfer, they added cyclosporin (CsH) to increase transduction by inhibiting lentiviral restriction factors.

First patient enrolled in NIH/NHLBI-sponsored Phase 1 Study of fostamatinib, Rigel's oral SYK inhibitor "Our Phase 1 study evaluating fostamatinib in patients with sickle cell disease is an ...