Canavan Disease Clinical Trial | ASPA Clinical Trial

AN INVESTIGATIONAL GENE THERAPY PROGRAM FOR CANAVAN DISEASE.

latest news

Aspa Therapeutics Presents Update on CANaspire Gene Therapy Trial for Canavan Disease at ESGCT 2024

Aspa Therapeutics is developing an investigational gene therapy to treat patients with Canavan disease. Aspa is the first company to initiate a multi-center, clinical trial for an investigational gene therapy for Canavan disease. There are two parts to Aspa’s program:

CANinform – a natural history studyjQuery("document").ready(function(){ toolTips('.tooltip_post_id_custom_c23387b576477e9919f7b7350a80d9ab','A study used to collect data about the course or impact of a disease in a group of patients\. In rare diseases it can be used to provide comparison data for a future investigational therapy instead of a placebo arm\. Regulators like the FDA can sometimes use natural history data to understand more about disease progression as they evaluate potential therapies for approval\.'); }); of Canavan disease to collect important data that may support the potential approval of the company’s gene therapy.

CANaspire – a clinical trial jQuery("document").ready(function(){ toolTips('.tooltip_post_id_custom_771ae06384b95ee374f3021c6929cd89','A research study of an investigational therapy that is closely monitored to protect participants and is designed to gather information about an investigational therapy such as its safety and potential benefits for patients\. Clinical trials are required before a potential treatment can be considered for approval by regulators such as the FDA or European Medicines Agency\.'); });designed to study the safety and potential effectiveness of an investigational gene therapy in Canavan patients.

ABOUT CANAVAN DISEASE

Canavan disease is a rare neurodegenerative disorder caused by mutations in the ASPA gene, which makes an essential protein – aspartoacylase – that breaks down a chemical compound called N-acetyl-L-aspartic acid (NAA). If NAA is not broken down it accumulates in a child’s brain and may prevent the proper formation of myelin. Myelin insulates the neurons, and without it they are unable to send messages properly.

ABOUT ASPA

BridgeBio founded Aspa with the sole purpose of developing a gene therapy to treat patients with Canavan disease and assembled a leadership team with extensive experience 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.