BridgeBio Pharma Inc. (Nasdaq: BBIO), a US-based commercial-stage biopharmaceutical company, announced on Wednesday that it has dosed its first patient in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy to treat Canavan disease, an ultra-rare and fatal disease that affects approximately 1,000 children in the United States and European Union, and for which there are currently no approved therapies.

The Phase 1/2 open-label study is aimed at assessing the safety, tolerability, and pharmacodynamic activity of the product in paediatric patients with Canavan disease. Each subject will receive a single intravenous (IV) infusion of BBP-812 in the initial dose-finding phase of the study.

The primary results of the study are safety, and change from baseline of urine and central nervous system N-acetylaspartate levels. The study will also assess motor function and development. An initial Phase 1/2 data readout for Canavan disease is expected in 2022.