Prosensa to Receive $1.5m from Charley’s Fund for Skipping of Exon 52 in Duchenne Muscular Dystrophy Patients
December 1, 2011
For immediate release
Leiden, The Netherlands, December 1, 2011 – Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics for rare diseases with unmet medical needs, announced today that it has received financing of $1.5m for three years from the charitable organisation Charley’s Fund, to support development of compounds for the skipping of exon 52 in patients with Duchenne Muscular Dystrophy (DMD).
Under the terms of the agreement, Prosensa will identify antisense oligonucleotides for the skipping of exon 52 in specific subpopulations of DMD patients. Prosensa will assess the safety and efficacy of the compounds in suitable preclinical models with the aim to move a selected candidate into clinical trials.
“Working closely with DMD patient advocacy groups has been central to Prosensa’s mission in identifying and developing effective treatments for DMD” commented Luc Dochez, Prosensa’s Chief Business Officer. “The company has already received financial support from Charley’s Fund and other groups during its early years. This additional support for the exon 52 program will enable us to accelerate our efforts towards clinical development.”
"Since our first investment in Prosensa six years ago, we have been impressed by their steadfast progress in drug development for DMD,” said Benjamin D. Seckler M.D., founder and President of Charley's Fund. “We are excited to partner once again to help more children with DMD benefit from this promising therapeutic strategy."
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About DMD
Duchenne Muscular Dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. Patients suffer from progressive loss of muscle strength due to the absence or defect of the dystrophin protein, often making them wheelchair bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.
About exon skipping
The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which lead to the manufacture of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the rest of the exons being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to a defective exon and thereby correct the reading frame, enabling the production of a novel dystrophin protein.
About Prosensa
Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics correcting gene expression in diseases with large unmet medical needs, in particular neuromuscular disorders. Prosensa’s current focus is on developing treatments for Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy and Huntington’s disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa’s lead compound (GSK2402968/PRO051), being developed by GSK, entered phase III clinical trials in January 2011.
Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, AGF Private Equity, GIMV, LSP and MedSciences Capital. For more information about Prosensa, please visit www.prosensa.com
About Charley's Fund
Charley’s Fund directs resources into the hands of researchers who have the best shot at developing a treatment or cure for Duchenne Muscular Dystrophy. Their goal is to cure DMD in time to save Charley’s life and the lives of thousands of boys like him. For more information about Charley’s Fund, please visit www.charleysfund.org
Prosensa recently won the 2011 Most Innovative Biotech SME Award by EuropaBio
Press contacts for this press release
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Luc Dochez - Chief Business Officer - Prosensa
languages: English, Dutch, French
Timezone: Europe/Amsterdam
Phonetel phone_1: +31 (0) 71 33 22 085
Phonetel phone_2: +31 (0) 71 33 22 100
E-mail: l.dochez@prosensa.nl
