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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:
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.
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.
