CANinform is a natural history study of patients with Canavan disease that will help researchers better understand the disease and provide a foundation for Aspa’s gene therapy program. The study is open to all individuals with a confirmed diagnosis of Canavan disease. Participating in CANinform is not required and does not offer any advantage for consideration for the treatment trial.
Medical records are a key source of natural history data and are needed to participate in CANinform. Aspa is offering a free record retrieval service to help families (outside of the EU) to obtain past medical records.
NAA is found mostly in neurons. If NAA is not broken down it accumulates in a child’s brain and may prevent the proper formation of myelin, the protective covering that helps neurons communicate with each other.
The deficits in myelin are thought to cause Canaan’s effects on movement, lanquage and vision.
Aspa’s investigational gene therapy is given in a single intravenous dose and aims to provide working copies of the
ASPA gene throughout a child’s body.
The treatment uses an AAV9 viral vector designed to deliver the functional ASPA gene throughout the body and the brain.
Once the functional ASPA gene is expressed, it may help restore myelin in the brain. This can potentially improve the course of Canavan disease by addressing the underlying genetic cause.