Aspa Therapeutics is focused on developing a gene therapy for Canavan disease, a rare genetic disorder caused by mutations in the ASPA gene, which leads to the accumulation of N-acetyl-L-aspartic acid (NAA) in the brain. This buildup is detrimental to myelin, the protective sheath around nerves, resulting in severe neurological symptoms and developmental delays in affected individuals. The company is utilizing adeno-associated virus (AAV) gene therapy to deliver functional copies of the ASPA gene to the brain, aiming to restore the breakdown of NAA and mitigate the disease's effects. This innovative approach has shown promise in preclinical models, demonstrating the potential to improve survival and motor function.

Aspa Therapeutics collaborates with the University of Massachusetts Medical School to advance its gene therapy program, which is currently in clinical trials. The company is committed to addressing the unmet medical needs of patients with Canavan disease, as there are no approved therapies available. By engaging with patient advocacy groups and the broader community, Aspa aims to keep families informed about the progress of their research and the development of potential treatments. The leadership team, comprised of experienced biotechnology professionals, is dedicated to bringing a safe and effective therapy to market as swiftly as possible.

United States

Aspa Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on developing a treatment for Canavan disease, a devastating inherited condition without an FDA-approved therapy. Aspa is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe and effective treatment for Canavan disease to patients as quickly as possible.

Aspa Therapeutics

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