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Article

Etuvetidigene Autotemcel: First Approval.

Molecular diagnosis & therapy - 1 May 2026

Blair Hannah A

Abstract excerpt

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...

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Identifiers

PMID: 41882319
DOI: 10.1007/s40291-026-00843-8

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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