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news.Sarepta Therapeutics and the University of Western Australia (UWA) have entered into an exclusive, worldwide licensing agreement for intellectual property rights to support the development of exon-skipping drug candidates for the treatment of Duchenne muscular dystrophy (DMD).
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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.
The agreement grants Sarepta rights to UWA’s extensive patent portfolio in DMD and enables it to build out its exon-skipping pipeline with new candidates based on its proprietary phosphorodiamidate morpholino oligomer (PMO) technology to address the majority of patients with the disorder worldwide.
Under the terms of the agreement, UWA is eligible to receive up to $7.1m in upfront and development milestone payments, as well as a low single-digit royalty on net sales of all approved medicines under the collaboration.
Sarepta president and CEO Chris Garabedian said that the agreement underscores the commitment to pursue treatments for all DMD patients who can benefit from exon-skipping technology, even those with rare genetic mutations.
UWA office of industry and innovation director Andy Sierakowski said, "This expanded collaboration with Sarepta enables us to translate our understanding of the dystrophin gene into additional potential exon-skipping therapeutics that address a majority of patients with DMD, representing a major contribution by the University of Western Australia to the field of genetic medicine."
The deal expands an agreement first signed in 2008, which supported the development of several exon-skipping drugs including eteplirsen, Sarepta’s lead clinical candidate for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.