According to a news source, CureDuchenne, a U.S. national nonprofit seeking a cure for Duchenne Muscular Dystrophy (DMD), will provide up to €5 million (US$7 million) to Prosensa Holding N.V. to support a number of the biotech company’s RNA-based therapeutics designed to address unmet medical needs for patients with rare genetic disorders. The organizations have been collaborating since 2004, and the latest infusion of convertible promissory notes is intended to accelerate the development of and patient access to DMD therapies. Located in the Netherlands, Prosensa is currently pursuing a new drug application filing with the U.S. Food and Drug Administration under its accelerated approval pathway for lead DMD therapy, PRO44. It expects to conduct a placebo-controlled trial of the drug in the United States in the first half of 2015.
CureDuchenne Founder and CEO Debra Miller said, “We are very encouraged by the pioneering progress that Prosensa has made in Duchenne and are committed to the long-standing collaboration we have with Prosensa to support the development of treatments for boys diagnosed with this rare and devastating genetic disease. This partnership underscores the important role that patient groups play in accelerating the research and development for Duchenne and other diseases.” See Prosensa Holding N.V. News Release, August 11, 2014.