Gene therapy is being extensively studied around the world, with approximately 3,500 patients having received AAV gene therapy in research and commercial settings. Gene therapies have been approved by regulators in Europe and the U.S., including two approved AAV gene therapies in the U.S., one of which is for a rare neurological condition in children. It is important to consider safety of any investigational therapy, and families should thoroughly discuss potential risks and benefits of any clinical trial with the study doctors. The American Society of Gene and Cell Therapy has additional gene therapy information here.
For CANaspire specifically, Aspa’s investigational gene therapy has been studied for safety and effectiveness in animal models but has not yet been studied in humans. The CANaspire clinical trial is designed to study safety and potential effectiveness of this investigational gene therapy in Canavan disease patients.