Delandistrogene moxeparvovec

Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy. It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells. It is an adeno-associated virus vector-based gene therapy that is given by intravenous infusion (slow injection into a vein).

The most commonly reported side effects include vomiting, nausea, acute liver injury, pyrexia (fever), and thrombocytopenia (abnormally low platelet count in the blood). Two patients may have died from acute liver failure. Delandistrogene moxeparvovec was approved for medical use in the United States in June 2023. It was developed by Sarepta Therapeutics, together with Roche, and is manufactured by Catalent.