March 16, 2026

The US and the European Union authorise gene therapy for Wiskott-Aldrich Syndrome

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In early 2026, both the European Union and the US Food and Drug Administration (FDA) gave a marketing authorisation to a gene therapy for Wiskott-Aldrich Syndrome (WAS), Waskyra, in patients aged 6 months and older (including adults).

Waskyra has been authorised for patients aged 6 months and older with WAS who have a mutation in the WAS gene for whom hematopoietic stem cell transplantation is appropriate, but have no compatible stem cell donor. The therapy is made from the stem cells that are collected from the patient’s blood. They are genetically modified so that they can produce a functional WAS protein and are transplanted back into the patient. The modified cells migrate to the bone marrow, where they start making healthy blood and immune cells that produce a functional WAS protein.

This is a positive step and the company that markets this therapy (Waskyra) needs to start conversations with national authorities or private insurances in the different countries to make this therapy available in the different countries.

For more information, please refer to the FDA article or the European Medicines Agency information.