AAVrh74

AAVrh74 is a recombinant adeno-associated virus (AAV) serotype originally isolated from rhesus macaques, widely utilized as a viral vector platform for gene therapy. It is characterized by a robust tropism for skeletal and cardiac muscle cells and the ability to achieve widespread systemic distribution following intravenous administration. AAVrh74 is the vector platform used in delandistrogene moxeparvovec (Elevidys), the first FDA-approved gene therapy for Duchenne muscular dystrophy (DMD). It is also the basis for several investigational therapies targeting various forms of limb-girdle muscular dystrophy (LGMD). A significant clinical advantage of AAVrh74 is its relatively low seroprevalence in the human population compared to other common serotypes like AAV2, which potentially allows for the treatment of a broader patient population with fewer pre-existing neutralizing antibodies.

Other names

Adeno-associated virus serotype rh74rhesus-derived AAV serotype 74