Prosensa receives more than € 3.2 million to develop a curative treatment for Duchenne Muscular Dystrophy

Leiden, The Netherlands, January 26, 2006 – Prosensa announces that it received more than € 3.2 million for the development of its lead product for the treatment of Duchenne Muscular Dystrophy (DMD). Funding parties are Charley’s Fund (USA), MedSciences Capital (the Netherlands), Association Française contre les Myopathies (AFM) (France), Aktion Benni & Co. (Germany), SenterNovem (the Netherlands), and CureDuchenne (USA).

The funding concerns clinical research in the field of DMD, a genetic lethal childhood’s disease with an incidence of approximately 1 in 3,500 newborn boys. Clinical signs of muscle weakness start as early as 2 years of age affecting all voluntary muscles. Treatment of patients with DMD primarily involves supportive treatments. No curative treatment is yet available that can re-establish the function of dystrophin, the protein lacking in DMD patients. Prosensa has currently a collaborative effort towards developing a curative treatment for DMD with the Leiden University Medical Center (LUMC).

Prosensa’s compounds can correct genetic diseases at the RNA level and are based on the use of short nucleotide chains able to modulate posttranscriptional processes. In isolated cells of DMD patients and a mouse model of DMD, this approach has been demonstrated to restore the production of a functional dystrophin protein. Preparation for a first clinical trial is currently in progress.

“Receiving this financial support from these parties has been very important and reinforces our approach to develop a treatment for DMD” said G. Platenburg, CEO of Prosensa.

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