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

Leiden, The Netherlands, March 18, 2004 – Prosensa and the LUMC have been granted a Technische Samenwerkings (TS) 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 technology development 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, led by Prof. Dr. G.J.B. van Ommen. A potential genetic therapy will be explored aimed at restoring the reading frame in muscle cells from DMD patients through targeted modulation of dystrophin pre-mRNA splicing using anti-sense compounds.

‘With the antisense approach we actually make use of how Nature turns the genetic code into cellular function. Only now we correct an error, and make functional protein from a defective gene. This subsidy will greatly help us to work as fast as we can to get this therapy operational in the coming five to ten years’, says Professor van Ommen.

‘We are extremely proud to have been awarded this grant. It will strengthen our collaboration with the LUMC and allow to expand our activities into developing muscle targeting technology needed to further move toward a viable cure for this terrible disease’, says Gerard Platenburg, Prosensa’s CEO.

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