{"title":"Etuvetidigene Autotemcel: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40291-026-00843-8","DOIUrl":null,"url":null,"abstract":"<p><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 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.</p>","PeriodicalId":49797,"journal":{"name":"Molecular Diagnosis & Therapy","volume":" ","pages":"539-543"},"PeriodicalIF":5.8000,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Diagnosis & Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40291-026-00843-8","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2026/3/25 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0
Abstract
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.
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Molecular Diagnosis & Therapy welcomes current opinion articles on emerging or contentious issues, comprehensive narrative reviews, systematic reviews (as outlined by the PRISMA statement), original research articles (including short communications) and letters to the editor. All manuscripts are subject to peer review by international experts.
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