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Sarepta Therapeutics, UCL partner on DMD drug target
January 2013

CAMBRIDGE, Mass.—Sarepta Therapeutics, a developer of innovative RNA-based therapeutics, announced last month a collaboration with University College London for the development of an 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 UCL scientist Prof. Francesco Muntoni, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from Europe and the United States.  
The program will be based on Sarepta's advance proprietary RNA-based platform, Phosphorodiamidate Morpholino Oligomers (PMOs), a backbone chemistry that provides enhanced target tissue specificity, increased potency to allow for more efficient dosing and greater uptake within a cell to further increase protein production. Targeting exon 53 with this technology will potentially address one of the most prevalent sets of mutations in DMD that are amenable to exon-skipping by restoring the cellular machinery's ability to produce a functional dystrophin protein.  
An 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.
"The initiation of this program, along with our other collaborations for exons 45 and 50, continues to advance Sarepta's strategy in pursuing exon-skipping therapeutics for all of the DMD patients who may benefit from this drug technology," stated Chris Garabedian, President and CEO of Sarepta Therapeutics. "Our goal of demonstrating that the success of eteplirsen 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."



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