BridgeBio Pharma Announces Early Positive Data for BBP-812, its Investigational AAV9 Gene Therapy for Canavan Disease

- These results are the first reported demonstration of rapid and robust treatment changes in key disease markers associated with the severity of disease  

- Initial pharmacodynamic results for two participants show unprecedented decreases in N-acetylaspartate (NAA) in the brain and urine, suggesting the therapy is producing functional ASPA enzyme 

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