Sarepta Therapeutics and University of Western Australia Announce Exclusive Worldwide Licensing Agreement for Exon-Skipping Program in Duchenne Muscular Dystrophy
04/11/13 8:30 AM EDT
"This agreement underscores our commitment to pursue treatments for all DMD patients who can benefit from our exon-skipping technology, even those with rare genetic mutations," said
"We are very encouraged by the recent progress of Sarepta's lead clinical program eteplirsen, which we believe demonstrates the strength of the underlying platform technology as well as our partner's drug development expertise in DMD," said
DMD is a rare and severe genetic disorder that affects boys and young men. It is associated with errors in the gene for dystrophin, a protein that plays a key structural role in muscle fibers. Patients with DMD lack functional dystrophin, and regular activity causes progressive muscle damage leading to weakness, loss of ambulation, respiratory and cardiac dysfunction, and eventually premature death. There are no approved treatments.
Exon-skipping is an innovative disease-modifying treatment approach designed to skip an exon in the dystrophin gene, thereby enabling the repair of specific genetic mutations and the production of a functional, but shorter, form of dystrophin. Sarepta currently has four exon-skipping programs in DMD addressing patients with genotypes amenable to skipping of exons 51, 45, 50 and 53.
Under the terms of the agreement, UWA is eligible to receive up to
About Duchenne Muscular Dystrophy
DMD is an X-linked rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs eventually spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.
Forward Looking Statement
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