Etuvetidigene Autotemcel for the Treatment of Wiskott-Aldrich Syndrome.
BACKGROUND Wiskott-Aldrich syndrome is a rare, X-linked, life-threatening inborn error of immunity and platelet disorder caused by variants in the gene WAS. Etuvetidigene autotemcel (etu-cel) is an autologous gene therapy consisting of hematopoietic stem and progenitor cells that have been transduced ex vivo with a len...