Adrenomyeloneuropathy is a debilitating lifelong disorder that currently has not treatments available. As an inherited disorder, gene correction is clearly necessary to alter the trajectory of disease burden. We have a developed an approach to deliver a healthy copy of the faulty gene directly into brain and spinal cord.

We have tested this in mice with the disease and have made key insights into a cell type critical to this process: neurons in the brain and alongside the spinal cord. With this knowledge we are now improving gene delivery using new viral vectors.

Importantly we have also developed industry partnerships that can help with manufacturing and setting up future clinical trials. Beyond creating a much-needed treatment, we are also through our studies gaining a better understanding of the disease biology of adrenomyeloneuropathy.

Project financed by ELA up to: 92 300 €