Delandistrogene moxeparvovec

DMD is a progressive, fatal X-linked recessive disease caused by mutations in the DMD gene that encodes dystrophin, an essential protein involved in muscle integrity and function. As a component of the dystrophin-glycoprotein complex (DGC), which absorbs shock and maintains muscle integrity during normal muscle contraction, dystrophin links the intracellular cytoskeleton network of muscle fibre cells to the sarcolemma. A lack of functional dystrophin protein results in the failure of DGC assembly, muscle inflammation and damage, impaired muscle fibre regeneration, and progressive and irreversible deterioration of muscle function and mass.

Delandistrogene moxeparvovec carries a transgene encoding microdystrophin. Microdystrophin is a shortened form of dystrophin and contains selected domains of dystrophin expressed in normal muscle cells. Microdystrophin delivered by delandistrogene moxeparvovec has been demonstrated to localize to the sarcolemma.