Prevail is developing AAV vectors carrying the gene that contains the instructions to produce the glucocerebrosidase enzyme.
Some people with genetic Parkinson’s disease have mutations in this gene. This causes the production of dysfunctional glucocerebrosidase, an important enzyme involved in lysosomal function - lysosomes break down molecules and cell components.
With this therapy, the company wants to introduce proper-functioning glucocerebrosidase enzymes into brain cells, so lysosomal function can be improved.
However, in clinical trials, severe adverse events were reported, like encephalitis (inflammation of the brain). It could be that the AAV vector causes too much inflammation in the brain.
