Duchenne Parent Project NL and United Parent Projects Muscular Dystrophy (UPPMD) grant subsidy to Prosensa for the collaboration with the LUMC on Duchenne Muscular Dystrophy

Leiden, The Netherlands, December 14, 2004 – Prosensa and the LUMC have been granted financial support from the United Parent Projects. Both DPP and UPPMD are 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.

‘The exon skip approach for DMD is very promising and the field is indeed moving towards clinical studies. The simplicity of the application of small synthetic RNA molecules combined with their specificity and efficacy is remarkable. After 15 years of complex gene therapy efforts, this is for many scientists a relief, and of course for DMD patients new hope.’ says Dr. Judith van Deutekom.

‘We are thrilled to participate in the exon skip Leiden clinical trial. For us, it means HOPE’, says Pat Furlong, president Parent Project Muscular Dystrophy.

‘We are extremely proud to have been awarded this grant. This support from the parent organizations is extremely important to us and strengthens our focus toward moving our technology into the next development phase. It will allow 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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