An Investigational Trial For Children With Canavan Disease

Canavan disease is a rare neurodegenerative disorder caused by mutations in the ASPA gene.


Patients are at the heart of Aspa's Clinical Program

Aspa Therapeutics is developing an investigational gene therapy to treat patients with Canavan disease, and has worked closely with the Canavan Foundation, Canavan Research Illinois, and National Tay-Sachs & Allied Diseases Association to advance its clinical program.

Aspa is the first company to initiate a multi-center, international clinical trial for an investigational gene therapy for Canavan disease.

The company has created this website to provide information about its clinical program. There are two parts to Aspa’s program:

CANinform – a natural history study of Canavan disease to collect important data that may support the potential approval of the company’s gene therapy.

CANaspire – a clinical trial designed to study the safety and potential effectiveness of an investigational gene therapy in Canavan patients.

Latest News

Aspa Therapeutics, a BridgeBio company, shared additional encouraging data from the CANaspire gene therapy trial. Details can be found here.

About Aspa

BridgeBio founded Aspa with the sole purpose of developing a safe and effective gene therapy to treat patients with Canavan disease, and its leadership team is experienced in developing therapies for rare diseases. 

Aspa is firmly committed to working with Canavan patient advocacy groups and families affected by Canavan disease throughout the development process.