BridgeBio: Early Positive Data for 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 

- If successful, BridgeBio’s gene therapy could be the first therapeutic option for children born with Canavan disease, a devastating and fatal neurodevelopmental disorder 

https://www.biospace.com/article/releases/bridgebio-pharma-announces-early-positive-data-for-bbp-812-its-investigational-aav9-gene-therapy-for-canavan-disease/