AAV9 gene therapy for Canavan disease
BBP-812 is an investigational adeno-associated virus (AAV) gene therapy for Canavan disease, which begins in infancy and progresses rapidly to severe neuromuscular symptoms and early mortality. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, correcting the disease. To date, results have shown promising pharmacodynamic, tolerability and preliminary functional efficacy. A broader Phase 1/2 data readout, including safety and efficacy findings and updates on the pharmacodynamic data, is expected in the first half of 2023.