Prosensa to Receive $1.5m from Charley’s Fund for Skipping of Exon 52 in Duchenne Muscular Dystrophy Patients

Leiden, The Netherlands, 1 December 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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