Israeli infant becomes first person treated with gene therapy for rare WWOX epilepsy

An eight-month-old baby in Israel has become the first person in the world to receive an experimental gene therapy for WOREE syndrome, a devastating inherited form of epilepsy, the Hebrew University of Jerusalem announced in June 2026. The treatment was given at Schneider Children's Medical Center in Petah Tikva under a compassionate-use framework, which allows an unapproved therapy to be used outside a formal trial when a patient's condition is severe and no alternatives exist.

WOREE syndrome is caused by faulty copies of the WWOX gene. Children with it typically suffer seizures that do not respond to medication, severe developmental impairment and a high risk of early death. The infant appeared healthy at birth but began having severe seizures at six weeks, and genetic testing confirmed the diagnosis. According to the university, the specific variant involved is especially common among people of Yemenite Jewish ancestry.

The therapy uses a harmless engineered virus, known as AAV9, to carry a working copy of WWOX directly into brain neurons through a single injection. It grew out of more than ten years of research by Prof. Rami Aqeilan at the Lautenberg Center for Immunology and Cancer Research at the Hebrew University's Faculty of Medicine. WWOX was first studied for its role in cancer, but his lab later showed that mice lacking it in the brain developed epilepsy, poor myelination and early death, and that a single dose of the gene therapy improved seizures, development and survival. The child was treated by Dr. Naama Orenstein and Dr. Dror Kraus at Schneider, and the US biotech company Mahzi Therapeutics licensed the technology and produced the clinical-grade material.

One month after the procedure the baby was stable and had been discharged, with no reported return of severe seizures, though the researchers stress that long-term follow-up is needed. Aqeilan told the Press Service of Israel that an application to the US Food and Drug Administration was expected within months and that, if trials go well, a medicine could be available within two or three years.