ASP2016

ASP2016 is an investigational **adeno-associated virus (AAV) gene therapy** developed to treat **Friedreich's Ataxia (FA)**, specifically targeting cardiac involvement. The therapy aims to restore functional expression of the **FXN gene** and increase frataxin protein levels in affected tissues, thereby correcting the underlying genetic deficit. It delivers a healthy copy of the FXN gene to patient cells via AAV8, with the goal of increasing frataxin levels and restoring normal cellular function. Administration is intravenous (IV), typically as a single infusion, and is accompanied by oral prednisolone to suppress immune reactions against the gene therapy[1][2][3][7][9].

Other names

ASP2016ASP-2016ASP 2016