Aspa Therapeutics has announced new data from the CANaspire gene therapy clinical trial

Aspa Therapeutics, a BridgeBio company, has announced new data from the CANaspire gene therapy clinical trial.

‘Decreases in N-acetylaspartate (NAA), a chemical marker in Canavan Disease, in the urine, cerebrospinal fluid (CSF), and the brain suggest the investigational treatment’s potential impact on this disease.’

Dr. Florian Eichler, lead investigator of the CANaspiretrial, noted:”To see this biochemical change suggests that we are reaching cells critical to the disease process, a milestone in this disease. The ongoing myelination seen on MRI and the new interactions witnessed between children and their parents are both encouraging.”

To read the press release: BridgeBio Pharma Announces Early Positive Data for BBP-812, its Investigational AAV9 Gene Therapy for Canavan Disease – bridgebiowp