Gene Therapy Etu-cel Linked to Sustained Benefit in Wiskott-Aldrich Syndrome
Pooled data from two prospective studies and an expanded-access program (27 participants total) evaluated etuvetidigene autotemcel (etu-cel), an autologous gene therapy, in patients with Wiskott-Aldrich syndrome. Over a median follow-up of 5.7 years, the study reported 96% overall survival at both 1 and 5 years, with one death, no evidence of insertional oncogenesis, and reductions in rates of severe infections and moderate-to-severe bleeding events compared with the year before treatment. The most common grade 3 or higher adverse event was central venous catheter-related infection.