CureDuchenne & Prosensa's Partnership Moves Forward, Results in Major Step Forward to Develop an Effective Therapy for Duchenne Muscular Dystrophy.

CORONA DEL MAR, Calif. -- Prosensa and Leiden University Medical Centre get approval for FIRST human trial of exon-skipping technology; DMD is most common and lethal childhood genetic disorder, afflicting one in 3,500 boys

CureDuchenne announced today that Prosensa, a Dutch biotech company, in conjunction with Leiden University Medical Centre (LUMC), has received approval to perform the first in-human trial using antisense ogligonucleotide, a "smart reagent" removing an unwanted segment of the faulty Duchenne Muscular Dystrophy (DMD) gene product. This trial represents a major step toward the development of an effective therapy for DMD, which is the most common and lethal genetic childhood disorder and which afflicts one in 3,500 boys.

CureDuchenne has partnered with Prosensa over the last two years to move their leading-edge science out of the labs and into clinical trials. "CureDuchenne puts dollars to work for immediate impact to save THIS generation of boys, truly giving them a chance for a lifetime," said Paul Miller, Secretary/Treasurer of CureDuchenne (and father of a Duchenne boy).

The exploratory study focuses 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. It will provide an important proof-of-principle for future studies based on systemic, full-body, delivery. Approval was granted from the Netherlands Central Committee on Research involving Human Subjects (CCMO) and the Medical Ethics Committee of LUMC.

"We are extremely glad to have permission for this study," said Gerard Platenburg, Prosensa's CEO. "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."

"We have spent five 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," said Dr. Judith van Deutekom. Also dedicated to this project are Dr. Jan Verschuuren and Prof./Dr. Gertjan van Ommen, respectively, lead investigators and Department head at the LUMC.

ABOUT DMD

DMD is a genetic lethal childhood's disease with an incidence of approximately 1 in 3,500 boys. Clinical signs of muscle weakness start as early as 2 years of age affecting all muscles. Treatment of patients with DMD to date primarily involves supportive treatments. No curative therapy, re-establishing the function of dystrophin, the protein that is lacking in DMD patients, is yet available.

ABOUT CUREDUCHENNE

CureDuchenne is a nonprofit organization founded in 2003 by Debra and Paul Miller. Their only son was diagnosed at the age of six with Duchenne, the most common and lethal childhood genetic disorder. "Our vision is our name...to cure Duchenne muscular dystrophy," said Debra Miller. CureDuchenne aggressively seeks out and funds research that will help THIS generation of Duchenne boys.

ABOUT PROSENSA

Prosensa BV is a young Dutch Biopharmaceutical Company focused on the discovery, development, and commercialisation of nucleic acid therapeutics correcting gene expression in diseases with large unmet medical needs, including neuromuscular disorders. In this work, Prosensa is working closely together with the research group of Dr. Judith van Deutekom in the LUMC Department of Human Genetics, head Prof. Dr. G.J.B. van Ommen and with the clinical group of Dr. Jan Verschuuren in the LUMC Department of Neurology, Head Prof. Dr. R. A.C. Roos. The group collaborates with Dr. Anneke van der Kooi van het Academisch Medisch Centrum in Amsterdam and Dr. Nathalie Goemans van het Universiteits Ziekenhuis Leuven in Belgie.

ABOUT LUMC

Leiden University Medical Centre (LUMC) aims to play a leading role nationally and internationally, in the continuous improvement of health care quality. LUMC's key tasks are research, patient care, and academic and post-academic medical education. It performs 11,500 daytime treatments and 19,000 hospital admissions yearly. It has 900 beds and employs 8700 people.