Gene therapy for FRDA is also under active investigation and studies of additive gene therapy using viral vectors carrying FXN cDNA have reported promising outcomes in vitro and in vivo. An adeno-associated virus (AAV)-based gene therapy product, LX2006, targeting FRDA cardiomyopathy, was recently approved for Phase 1/2 clinical trial by the United States Food & Drug Administration (FDA) following promising preclinical studies (Salami et al., 2020). The gene discussed is FXN; the disease is Friedreich ataxia.