Gene therapy etu-cel shows sustained benefit in Wiskott-Aldrich syndrome
Pooled data from two prospective studies and an expanded-access program (27 total participants) evaluated etuvetidigene autotemcel, an autologous lentiviral gene therapy, in patients with Wiskott-Aldrich syndrome. Over a median follow-up of 5.7 years, overall survival was 96% at both 1 and 5 years, with one death reported, and rates of severe infections and moderate-to-severe bleeding events decreased compared with the year before treatment; no insertional oncogenesis was observed, and the most common grade 3 or higher adverse event was central venous catheter-related infection. The authors report these findings as consistent with sustained clinical benefit.