Prosensa and LUMC get approval for in-human trial for ‘smart therapy’ to cure Duchenne Muscular Dystrophy

Leiden, The Netherlands, May 10, 2006 – On May 8, 2006 the Netherlands Central Committee on Research involving Human Subjects (CCMO) and the Medical Ethics Committee (IRB) of LUMC have granted Prosensa and LUMC permission to perform an exploratory study on the efficacy, safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide to restore production of the dystrophin, the protein defective in Duchenne Muscular Dystrophy (DMD) patients.

This very first in-human trial using antisense oligonucleotide, a ‘smart reagent’ removing an unwanted segment of the faulty DMD gene product, represents a major step toward the development of an effective therapy for DMD. It will provide an important proof-of-principle for future studies based on systemic, full-body, delivery.

"We are extremely glad to have permission for this study. We hope to start next month and expect to provide further information on the development in due time. We have all reasons to expect that this trial will prove the therapeutic potential of our technology, and thus form the basis for a viable cure for this terrible disease", says Gerard Platenburg, Prosensa's CEO.

“We have spent 5 years of laboratory and animal research and have successfully completed all the preparatory steps. Now the moment has come to see whether our vision to restore the defective protein has a chance of working in patients” say dr Judith van Deutekom, dr Jan Verschuuren and prof. dr Gertjan van Ommen, respectively lead investigators and Department head at the LUMC.

link to this press release