Texas Children’s Hospital Successfully Treats AADC Deficiency with First FDA-Approved AAV Gene Therapy

HOUSTON, Aug 19, 2025 – Texas Children’s Hospital announced a major medical milestone, successfully treating a three-year-old girl with the first-ever FDA-approved AAV gene therapy for Aromatic l-amino acid decarboxylase (AADC) deficiency. This rare, inherited neurological disorder prevents the brain from producing essential chemicals like dopamine and serotonin, which control movement and mood. Historically, a diagnosis of AADC deficiency had a life expectancy of just five to seven years.

The approved drug, Kebilidi (eladocagene exuparvovec-tneq), is a modified AAV2 vector-based gene therapy administered directly into the putamen area of the brain. The minimally invasive neurosurgery was performed by Dr. Daniel J. Curry, Director of Functional Neurosurgery and Epilepsy Surgery at Texas Children’s Hospital.

“Before now, AADC deficiency was a hopeless diagnosis,” said Dr. Curry. “With this treatment, we’ve entered a whole new era where we can deliver solutions to formerly untreatable genetic problems. This is the first step in hopefully many future strides toward the molecular correction of inborn deficits for which there used to be no cure.”

The young patient, who was diagnosed at 18 months old, had been experiencing poor muscle control and developmental delays. The treatment was administered without complications. After two weeks of hospital monitoring, she has returned home and is already showing signs of increased energy and happiness. While the full effects of the therapy are expected to take several months, the family is optimistic about her future.

Texas Children’s Hospital was the largest contributor to the clinical trial that led to the drug’s approval. Dr. Curry’s team is also investigating gene therapy for other neurodegenerative diseases like Rett syndrome and NGLY-1 deficiency, with the hope of not only treating the root cause of these conditions but potentially reversing their effects.