Aspa is developing a gene therapy to treat Canavan disease, and has both short and long term goals for its clinical program. The CANinform natural history study is a crucial first step in working towards a treatment trial. The gene therapy treatment trial will build on this natural history data, and will use a therapeutic approach different from previous gene therapy trials in Canavan disease.
Aspa’s gene therapy uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body and into the brain. The goal of this gene therapy is to address the underlying cause of Canavan disease and help improve signs and symptoms of the disease. AAV9 gene therapy has been extensively studied in other rare diseases, including the recent approval of the first AAV9 gene therapy for a pediatric neurodegenerative disease.