Prosensa Awarded $200,000 Research Grant from PPMD

Leiden, The Netherlands, June 27, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ:RNA), the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, today announced that Dr. Judith van Deutekom, Prosensa’s Vice President of Drug Discovery, has been award­ed a $200,000 research grant from Parent Project Muscular Dystrophy (PPMD), the largest non-prof­it orga­ni­za­tion in the United States focused on find­ing a cure for Duchenne Muscular Dystrophy (DMD), to fund the devel­op­ment of a mul­ti exon-skip­ping strat­e­gy for exons 10-30 of the dys­trophin gene.
The project titled “Single AON-induced mul­ti­ple exon skip­ping in the mdx mouse mod­el” will focus on obtain­ing proof-of-con­cept in the mdx mouse mod­el. The stud­ies are expect­ed to be com­plet­ed in the sec­ond half of 2015.
“We are delight­ed with PPMD’s fund­ing of our project that aims at the pre-clin­i­cal devel­op­ment of sin­gle anti­sense oligonu­cleotides capa­ble of induc­ing mul­ti­ple exon skip­ping in the exon 10 to 30 region of the DMD gene,” com­ment­ed Dr. van Deutekom. “This would apply to DMD patients with more rare muta­tions upstream of the dele­tion hot spot (exons 45-55), which could address up to 13% of DMD patients.”
“Recognizing the need to bring this promis­ing tech­nol­o­gy to as many with Duchenne as pos­si­ble, in August 2013 we announced a request for appli­ca­tions focused on the devel­op­ment of anti­sense oligonu­cleotides for muta­tions in the dys­trophin gene that were not already in clin­i­cal devel­op­ment. Our Scientific Advisory Committee, com­posed of lead­ing experts in the field, select­ed the pro­pos­al from Prosensa as one of the appli­ca­tions to be fund­ed,” said Sharon Hesterlee, PPMD’s Vice President of Research. “We very much look for­ward to work­ing with Prosensa to sup­port impor­tant clin­i­cal research ini­tia­tives that could have a sig­nif­i­cant impact on the health and lives of young boys diag­nosed with this rare and dev­as­tat­ing genet­ic dis­ease,” she added.

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