FDA ApprovedUnited States
First FDA-Approved Gene Therapy for Wiskott-Aldrich Syndrome
Waskyra (etuvetidigene autotemcel) — one-time gene-modified stem-cell therapy for Wiskott-Aldrich syndrome when no matched related donor is available
FDA approval on 9 December 2025 for Waskyra (etuvetidigene autotemcel) suspension — an autologous hematopoietic stem-cell gene therapy given as a one-time IV…
This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need a transplant-style path and do not have a suitable…