Etuvetidigene autotemcel

Etuvetidigene autotemcel, sold under the brand name Waskyra, is a gene therapy medication used for the treatment of Wiskott–Aldrich syndrome.

The most common side effects include rash, respiratory tract infection, febrile neutropenia, catheter related infection, vomiting, diarrhea, liver injury, and petechiae.

The active substance of etuvetidigene autotemcel consists of genetically modified autologous CD34+ hematopoietic stem cell enriched population transduced ex vivo with a lentiviral vector encoding the human Wiskott–Aldrich syndrome gene. Etuvetidigene autotemcel is a gene therapy that inserts the Wiskott–Aldrich syndrome corrected gene into the cell's genome, making the genetically modified cells capable of expressing the functional Wiskott–Aldrich syndrome protein. The genetically modified cells engraft and repopulate the hematopoietic compartment. They differentiate and produce biologically active lymphoid and myeloid progenitors whose progeny express Wiskott–Aldrich syndrome protein.

Etuvetidigene autotemcel was approved for medical use in the United States in December 2025, and in the European Union in January 2026.