rAAV-Olig001-ASPA
Gene TherapyrAAV-Olig001-ASPA is an investigational gene therapy specifically designed to treat Canavan disease, a severe childhood genetic disorder characterized by the degeneration of white matter in the brain.
- Utilizes proprietary adeno-associated virus (AAV) vector technology
- Delivers a functional copy of the ASPA gene directly to oligodendrocytes
- Administered via intracerebroventricular injection for effective brain cell targeting
- Early trials showing reductions in disease biomarkers and improvements in white matter