AAVrh.10hFXN-HA

AAVrh.10hFXN-HA is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of the neurologic manifestations of Friedreich's ataxia (FA). It utilizes the AAVrh.10 serotype to deliver a functional human frataxin (FXN) coding sequence, which is tagged with hemagglutinin (HA) for research tracking. The therapy aims to restore frataxin levels in the cerebellum to address ataxia and gait instability. Research conducted at Weill Cornell Medicine has explored various delivery routes, including direct intraparenchymal cerebellar administration and intracisternal administration via the cisterna magna, to optimize vector distribution and overcome dose-limiting toxicities and pre-existing immunity.