Month: September 2024

Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease (CANaspire Clinical Trial) [https://treatcanavan.com/canaspire-trial/]   Aspa’s investigational gene therapy trial uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body, including into the brain, to address the underlying cause of Canavan disease. The AAV9 …

Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease Read More »

Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease (CANinform Study) [https://clinicaltrials.gov/study/NCT04126005] 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 individuals with a confirmed diagnosis of Canavan disease.