Sarepta Announces Collaboration for Development of Added Exon-Skipping Product for DMD

Sarepta Therapeutics SRPT announced today a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in development, in support of Sarepta's broad-based program for the treatment of Duchenne muscular dystrophy (DMD). Sarepta's collaboration is with University College London's (UCL) scientist, Professor Francesco Muntoni, MD, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the EU and US. The EU Health Innovation-1 2012 Collaborative research grant will support certain IND-enabling activities and clinical proof of concept studies for an exon 53-skipping therapeutic. Sarepta recently announced positive results from its extension study of its Phase IIb trial of eteplirsen, its exon 51-skipping therapeutic candidate for the treatment of DMD. Sarepta is also developing other PMO-based exon-skipping drug candidates for exons 45 and 50.