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<p>Etuvetidigene autotemcel (WASKYRA<sup>™</sup>) is an autologous haematopoietic stem cell (HSC)-based gene therapy being developed by Fondazione Telethon for the treatment of Wiskott–Aldrich syndrome (WAS). In December 2025, etuvetidigene autotemcel was approved in the USA for the treatment of paediatric patients aged 6&#xa0;months and older and adults with WAS who have a mutation in the <i>WAS</i> gene for whom HSC transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. Etuvetidigene autotemcel was approved in the EU for the same indication in January 2026. This article summarizes the milestones in the development of etuvetidigene autotemcel leading to this first approval for the treatment of WAS.</p>

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Etuvetidigene Autotemcel: First Approval

  • 期刊论文

  • 发表日期：

    2026年3月25日

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  • Hannah A. Blair

摘要

Etuvetidigene autotemcel (WASKYRA™) is an autologous haematopoietic stem cell (HSC)-based gene therapy being developed by Fondazione Telethon for the treatment of Wiskott–Aldrich syndrome (WAS). In December 2025, etuvetidigene autotemcel was approved in the USA for the treatment of paediatric patients aged 6 months and older and adults with WAS who have a mutation in the WAS gene for whom HSC transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. Etuvetidigene autotemcel was approved in the EU for the same indication in January 2026. This article summarizes the milestones in the development of etuvetidigene autotemcel leading to this first approval for the treatment of WAS.