Prosensa Awarded $200,000 Research Grant from PPMD

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.  About Prosensa Holding N.V.  Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, Myotonic dystrophy and Huntington's  About DMD  Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 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. As a result, patients suffer from progressive loss of muscle strength, often rendering 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 segments of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot be processed past the fault. This prevents the remainder of the exons from 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, or containing, the fault and thereby correct the RNA processing, enabling the production of a novel, largely functional dystrophin protein. Prosensa's exon skipping technology was licensed from Leiden University Medical Center.  About PPMD  Parent Project Muscular Dystrophy (PPMD) is a not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization's mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion. Only this comprehensive approach will lead to the day that 100% of those diagnosed can turn to a treatment that will end Duchenne. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information,  Forward Looking Statement  This  press  release   contains  certain   forward-looking  statements.   All  statements, other than statements of historical facts, contained in this press release, including  statements  regarding  our  strategy,  future  operations,  future financial position, future revenues, projected costs, prospects,  plans  and objectives  of  management,  are  forward-looking  statements.  The  words  "anticipate,"  "believe,"  "estimate,"  "expect,"  "intend,"  "may,"   "plan,"  "predict,"  "project,"  "target,"   "potential,"  "will,"  "would,"   "could,"  "should,"  "continue,"  and  similar  expressions  are  intended  to  identify  forward-looking  statements,  although  not  all  forward-looking   statements  contain these  identifying words.  Forward-looking  statements in  this  press  release include statements around  our multi-exon skipping program,  PROSPECT,  and grant funding around  this program. Actual  results may differ  materially  from those  projected or  implied in  such forward-looking  statements.  Such  forward-looking  information  involves  risks  and  uncertainties  that  could  significantly affect  expected results.  These  risks and  uncertainties  are  discussed in the  Company's SEC filings,  including, but not  limited to,  the  Company's Form 6-K containing this press  release and certain sections of  the  Company's  Registration   Statement   on   Form   20-F.   In   addition,   any  forward-looking statements represent our views only as of today and should not be relied upon as representing our views  as of any subsequent date. While  we  may elect to  update these  forward-looking statements  at some  point in  the  future, we specifically disclaim  any obligation to do  so, even if our  views  change.  CONTACT: Prosensa Holding N.V.          Celia Economides, Senior Director, IR & Corporate Communications  Prosensa Holding B.V. Logo  
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