CureDuchenne to fund Prosensa for the pre-clinical and clinical development of a potential Duchenne Muscular Dystrophy therapy

Leiden, The Netherlands, December 18, 2004 – Prosensa will receive financial support from CureDuchenne. CureDuchenne is a not-for-profit organizations dedicated to the development of a therapy for Duchenne muscular dystrophy.

The grant concerns (pre-) clinical development in the field of Duchenne Muscular Dystrophy (DMD). DMD is one of the most common lethal genetic disorders, affecting all populations worldwide. It is the most devastating of the muscular dystrophies. It is caused by mutations in the DMD gene that abolish the synthesis of a functional dystrophin protein. No known cure is presently available. Treatment is focused to improve quality of life and includes artificial respiration and orthopedic surgery to correct skeletal deformation, which results from muscle weakness. Pharmacological treatment options are very limited. Prosensa has currently a collaborative effort towards developing a therapy for Duchenne Muscular Dystrophy with the group of Dr Judith van Deutekom in the Department of Human Genetics, LUMC, chaired by Prof. Dr. G.J.B. van Ommen. A novel genetic therapy is explored aimed at inducing the skipping of specific exons during the splicing of the gene's pre-mRNA in cultured muscle cells from DMD patients using small synthetic RNA molecules. This exon skipping restores the genetic open reading frame, and thus facilitates the synthesis of modified but largely functional dystrophin.

‘After almost a year’s investigation, we found exon skipping to hold the most near term promise for Duchenne, and Prosensa is poised to move this technology forward’, says Debra Miller, president CureDuchenne.

‘We are extremely proud to have been awarded this grant. This support from CureDuchenne strengthens our focus toward moving our technology into the next development phase. It will allow us to expand our activities into clinical development of our technology needed to further move toward a viable cure for this terrible disease’, says Gerard Platenburg, Prosensa’s CEO.

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