New Gene Therapy Vector Technology for Canavan Treatment


The rAAV-Olig001-ASPA gene therapy is given as a one-time administration into fluid-filled cavities of the brain, called ventricles, and hence, the route of administration is called intracerebroventricular. This process enables the rAAV-Olig001-ASPA gene therapy to directly target the oligodendrocytes of the brain – the cells affected in Canavan disease and where the functional copy of ASPA gene needs to be delivered.

Canavan disease is an ideal candidate for this new technology because it is based on a single target, the ASPA gene, which codes for an enzyme responsible for breaking down NAA into acetate and aspartate. The ASPA gene mutation alters the activity of the aspartoacylase enzyme, resulting in an accumulation of NAA and depletion of acetate and aspartate that ultimately results in failure of brain growth and development.

Preliminary results show that patients treated in the Phase 1/2 First-in-Human clinical trial for Canavan disease have shown reductions in NAA levels, increases in white matter and myelin, and patient improvements within six months of treatment.