The company partnered with Francesco Muntoni, professor of pediatric neurology and head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London, and other EU and US scientists.
Collaboration focuses on developing a drug that skips another sequence of DNA known as exon 53, to deal with a genetic mutation believed to cause DMD.
Sarepta Therapeutics president and CEO Chris Garabedian said commencement of the development program advances the company’s approach in pursuing exon-skipping therapeutics for DMD patients.
"Our goal of demonstrating that the success of eterplirsen can be reproduced across other exon-skipping targets is a critical step in being able to treat more boys and young men affected with this devastating disease," Garabedian added.
The exon 53-skipping drug is a fourth drug candidate in the development of DMD medication by the company that leverages Sarepta’s proprietary RNA-based platform, Phosphorodiamidate Morpholino Oligomers (PMOs).
Few IND-enabling activities and clinical proof of concept studies of the new therapeutic will be supported by the EU Health Innovation-1 2012 Collaborative research grant.
Positive results from Phase IIb DMD trial of eteplirsen, an exon 51-skipping therapeutic, were earlier reported while other PMO-based exon-skipping candidates targeting exons 45 and 50 are also being developed by the company.