FDA Approves Two Gene Therapies for Inherited Immune Disorders, Highlighting Access Challenges
Within the past year, the FDA has approved two gene therapies for inherited immune disorders: etuvetidigene autotemcel (etu-cel, Waskyra) for Wiskott-Aldrich syndrome (WAS) and Exagamglogene autotemcel (Casgevy) for sickle cell disease and transfusion-dependent β-thalassemia. Etu-cel, approved in December 2025, is for WAS patients aged 6 months and older who are eligible for hematopoietic stem cell transplantation but lack a suitable donor. Casgevy's eligibility was expanded in July 2026 to include patients aged 2 years and older with sickle cell disease and recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia, lowering the previous age threshold. While these approvals offer new treatment options, experts at the 2026 American Society for Transplantation and Cellular Therapy Gene Therapy Summit emphasized ongoing challenges in manufacturing quality, long-term safety, and patient access. For instance, Casgevy's label requires lifelong malignancy surveillance and regular blood counts, highli...
