Prosensa Holding N.V., the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, 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,” said 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.
Date: June 27, 2014
Filed Under: Drug Discovery