There are challenges to delivering viral gene therapies. They can elicit an immune response, especially if the need to dose more than once arises due to the complexities of a disease.
In addition, appropriate distribution in the nervous system of CNS-specific therapies is technically challenging.
We are addressing those challenges by advancing a novel AAV dosing platform that enables:
- Redosing by subverting the immune response
- Delivery through the vagus nerve enabling broad coverage of the nervous system
We believe that direct administration of our AAV9 therapies to the vagus nerve could be useful to treat the peripheral and autonomic manifestations of the CNS diseases in our pipeline.