Gene therapy shows sustained benefit in Wiskott-Aldrich syndrome

Pooled data from two prospective studies and an expanded-access program (27 participants total) evaluated etuvetidigene autotemcel, an autologous lentiviral gene therapy, in 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, and the rate of severe infections and moderate-to-severe bleeding events per person-year both decreased substantially compared with the year before treatment; the most common grade 3 or higher adverse event was central venous catheter-related infection, and no insertional oncogenesis was observed. The study authors report these results as consistent with sustained clinical benefit.

Most relevant for: Patients with Wiskott-Aldrich syndrome, including infants and young children undergoing evaluation for gene therapy.

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