Sarepta to develop new exon 53 skipping drug for Duchenne muscular dystrophy

27th November 2012 (Last Updated November 27th, 2012 18:30)

Sarepta Therapeutics has entered into a research partnership to develop an exon 53-targeted exon-skipping drug for the treatment of Duchenne muscular dystrophy (DMD).

Sarepta Therapeutics has entered into a research partnership to develop an exon 53-targeted exon-skipping drug for the treatment of Duchenne muscular dystrophy (DMD).

The company has collaborated with Francesco Muntoni, professor of paediatric neurology and head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London, and other EU and US scientists for the development of its fourth drug for the treatment of DMD.

Muntoni said the recent clinical data on eteplirsen-targeting exon 51 is a good foundation for using the company's technology against exon 53.

"We are pleased to be working with Sarepta to bring this exciting exon-skipping therapeutic approach to Europe and the broader DMD population," Muntoni said.

Exon 53 causes a genetic mutation that results in DMD.

"Exon 53 causes a genetic mutation that results in DMD."

IND-enabling activities and clinical proof-of-concept studies for an exon 53-skipping therapeutic will be carried out using the EU Health Innovation-1 2012 Collaborative research grant.

Company's DMD discovery programme is based on its proprietary RNA-based platform, Phosphorodiamidate Morpholino Oligomers (PMOs) that enables improved target tissue specificity, amplified potency to allow for efficient dosing, and superior uptake within a cell to further increase protein production.

Sarepta Therapeutics president and CEO Chris Garabedian said starting the development programme 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 said.

Earlier, positive results from a Phase IIb DMD extension study of exon 51-skipping therapeutic candidate, eteplirsen, were revealed while PMO-based exon-skipping drug candidates for exons 45 and 50 are currently being developed.