Motor Neuron Disease (MND/ALS)

In MND/ALS the motor neurons that carry signals for voluntary movement progressively degenerate, along with the glial cells that support them. GDNF acts on both.

Innervate delivers AAV5–GDNF to the motor cortex, where it is taken up and carried by axonal transport to secondary motor neurons in the brainstem and spinal cord. Pre-clinically, this markedly increases GDNF across the motor cortex and, through that transport, the brainstem and spinal cord — reaching the cells that matter most while confining the therapy to CNS targets.

By protecting neurons from neurotoxic glutamate, supporting the surrounding astrocytes and oligodendrocytes, and sustaining myelination and metabolic support, GDNF addresses the degeneration and dysfunction at the heart of the disease.

MND/ALS is Innervate’s lead programme. First-in-man studies are planned at the John Radcliffe Hospital in Oxford, led by Prof. Kevin Talbot and Prof. Martin Turner.