The first intrathecal administration of an adeno-associated virus (AAV) gene therapy vector is underway at NIH for the rare neurodegenerative disorder, giant axonal neuropathy (GAN), a differential diagnosis of Charcot-Marie-Tooth Type 2. To date, two subjects have been injected with self-complimentary AAV serotype 9/GAN. There have been no adverse events.
If this IND is successful at achieving broad distribution throughout the CNS, it could be an answer to monogenic loss of function disorders whose targets are upper and lower motor neurons, as well as dorsal root ganglia.
All pre-clinical work for this investigational biologic was funded by Hannah’s Hope Fund for GAN, a 501c3 public charity. More about this trial.