Sarepta Therapeutics, a developer of innovative RNA-based therapeutics, has entered into a collaboration with the 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 for the development of an additional exon-skipping drug targeting exon 53. This is the fourth drug in development, in support of Sarepta's broad-based programme for the treatment of Duchenne muscular dystrophy (DMD)

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.

"The recent compelling clinical data on eteplirsen targeting exon 51, which started with our work on the Phase I study in the UK, provides a strong foundation for using Sarepta's technology against exon 53," said Francesco Muntoni, professor of pediatric neurology and head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London. "We are pleased to be working with Sarepta to bring this exciting exon-skipping therapeutic approach to Europe and the broader DMD population."

This programme will be based on Sarepta's advance proprietary RNA-based platform, Phosphorodiamidate Morpholino Oligomers (PMOs), which is a novel 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 (deletion of exons 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, or 52) by restoring the cellular machinery's ability to produce a functional dystrophin protein.

"The initiation of this programme, 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," said Chris Garabedian, president and CEO of Sarepta Therapeutics."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."

The aim of the EU investment in health research is to improve the health of European citizens, to address global health issues and to boost the competitiveness of European health-related industries. Each year the European Commission publishes calls for proposals to which researchers can apply for collaborative research grants.

The European Commission implements a thorough and transparent evaluation process to identify proposals worthy of funding. The process is firmly grounded in the principle of access for all and the codes of fairness, impartiality, confidentiality, efficiency and ethics.

The Dubowitz Neuromuscular Centre (DNC) provides a multidisciplinary service as a leading clinical and research center specializing in neuromuscular disorders affecting individuals in the pediatric age. The DNC provides clinical assessment, diagnostic services, advice on treatment and rehabilitation, and is involved in clinical trials and in basic research focused on understanding the cause of neuromuscular diseases in childhood, and identifying novel therapeutic intervention.Excellence in this field is demonstrated not only by the National Commissioning Group (NCG) status achieved in 2000 to provide a national specialist diagnostic and assessment service for Congenital Muscular Dystrophies and Congenital Myopathies, but also by the designation as a muscle centre by the Muscular Dystrophy Campaign.

The DNC is part of the UCL Institute of Child Health and Great Ormond Street NHS Trust and is a member of the recently created MRC Neuromuscular Translational Research Centre at UCL. In addition to the service provided for all paediatric neuromuscular disorders, the DNC is involved with clinical, molecular and cell biological research mostly focused on the genetic basis of congenital muscular dystrophies; on muscle stem cell and on experimental therapies for Duchenne muscular dystrophy.

Eteplirsen is Sarepta's lead drug candidate that is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression. Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to improve, stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.