Senter grants IS subsidy to Prosensa for the collaboration with the LUMC on Duchenne Muscular Dystrophy

Leiden, The Netherlands, December 20, 2004 – Prosensa and the LUMC have been granted an Innovation Samenwerkings (IS) grant from Senter. Senter is affiliated to the Ministry of Economic affairs and is responsible for the execution of subsidy, credit and fiscal programs in the field of technology, energy, environment and international collaboration. The Technological grant is a general grants scheme to stimulate R&D co-operation. The projects are appraised and prioritised based on the criteria of co-operation, technological innovation and economic perspective.

The grant concerns clinical research in the field of Duchenne Muscular Dystrophy. Duchenne is the most common lethal genetic disorder, affecting all populations worldwide. It is the most common and most devastating of the muscular dystrophies. 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.

‘We are extremely happy to have been awarded this grant. 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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