The U.S. Food and Drug Administration (FDA) has approved Waskyra, the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
The therapy represents a major scientific and clinical achievement, offering new hope for patients affected by this condition. WAS, seen almost exclusively in males, affects blood cells and cells of the immune system. Until now, treatment options for patients with WAS have been limited to symptomatic management and allogeneic hematopoietic stem cell transplantation, which is most effective early in life and only possible with matched donors. Waskyra restores functional WAS protein expression in affected cells, addressing the underlying cause of the disease.