Sarepta Therapeutics, UCL partner on DMD drug target

CAMBRIDGE, Mass.—Sarepta Therapeutics, a developer ofinnovative RNA-based therapeutics, announced last month a collaboration withUniversity College London for the development of an exon-skipping drugtargeting exon 53, its fourth drug in development, in support of Sarepta'sbroad-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 ChildHealth and other scientists from Europe and the United States.

The program will be based on Sarepta's advance proprietaryRNA-based platform, Phosphorodiamidate Morpholino Oligomers (PMOs), a backbonechemistry that provides enhanced target tissue specificity, increased potencyto allow for more efficient dosing and greater uptake within a cell to furtherincrease protein production. Targeting exon 53 with this technology willpotentially address one of the most prevalent sets of mutations in DMD that areamenable to exon-skipping by restoring the cellular machinery's ability toproduce a functional dystrophin protein.

An EU Health Innovation-1 2012 Collaborative research grantwill support certain IND-enabling activities and clinical proof-of-conceptstudies for an exon 53-skipping therapeutic. Sarepta recently announcedpositive results from its extension study of its Phase IIb trial of eteplirsen,its exon 51-skipping therapeutic candidate for the treatment of DMD. Sarepta isalso developing other PMO-based exon-skipping drug candidates for exons 45 and50.

"The initiation of this program, along with our othercollaborations for exons 45 and 50, continues to advance Sarepta's strategy inpursuing exon-skipping therapeutics for all of the DMD patients who may benefitfrom this drug technology," stated Chris Garabedian, President and CEO ofSarepta Therapeutics. "Our goal of demonstrating that the success of eteplirsencan be reproduced across other exon-skipping targets is a critical step inbeing able to treat more boys and young men affected with this devastatingdisease."