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EMA – First gene therapy to treat rare disease Wiskott-Aldrich syndrome

EMA has recommended granting a marketing authorisation in the European Union (EU) for Waskyra (etuvetidigene autotemcel) to treat people aged 6 months and older with Wiskott-Aldrich syndrome (WAS) who have a mutation in the WAS gene. Waskyra is used to treat patients for whom a haematopoietic stem cell transplantation (HSCT) is appropriate, but for whom no suitable stem cell donor is available.

Wiskott-Aldrich syndrome is a rare, inherited disease, seen almost exclusively in males, that affects blood cells and cells of the immune system (the body’s natural defences). It is caused by abnormalities in the gene that produces the WAS protein found in blood cells and certain immune cells. Because people with the condition lack a functional WAS protein, their immune cells and blood cells do not develop and function normally. […]

Waskyra is a gene therapy medicine that is made from stem cells (called CD34+ cells) that are collected from the patient’s blood. The cells are genetically modified in the laboratory so that they can produce a functional WAS protein. When transplanted back into the patient after a conditioning (preparatory) regimen, the modified cells migrate to the bone marrow where they start making healthy blood and immune cells that produce a functional WAS protein, thereby helping to relieve the symptoms of the disease. Waskyra is given only once, by infusion (drip) into a vein.

More information on the EMA Website.


OpenEdition vous propose de citer ce billet de la manière suivante :
vroby (11 décembre 2025). EMA – First gene therapy to treat rare disease Wiskott-Aldrich syndrome. ELSIBI. Consulté le 19 février 2026 à l’adresse https://doi.org/10.58079/15bv1


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