GT-FA is a recombinant, adeno-associated virus, containing the transgene that encodes for the protein frataxin (FXN) that is currently being developed for the treatment of Friedreich ataxia (FA). The goal of this therapy is to increase the amount of frataxin levels in the central nervous system.  By restoring the level of FXN, GT-FA has the potential to improve both physical and cognitive abilities in patients with FA, including the improvement of balance, sensory capability, strength, cognition, and coordination.  Currently, there are no therapies available that address the underlying issue of FA. 

GT-FA is in pre-clinical development and has been associated with the restoration of FXN levels in multiple models of disease. 

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