Company Profile

Prosensa is a biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM1), Huntington's Disease (HD) and Spinal Muscular Atrophy (SMA).

The company has developed a portfolio of clinical and pre-clinical RNA-based drug candidates. Prosensa’s current clinical portfolio is focused on the treatment of Duchenne Muscular Dystrophy. Its lead product, PRO051/GSK2402968, aims at restoring dystrophin expression and improving muscle condition and function in a relatively large subpopulation of Duchenne patients and has successfully completed a Phase I/II study. Preparations are ongoing to start enrolling patients in a Phase III study in 2010. PRO044, the company’s second product candidate, addresses another subpopulation of Duchenne patients and has entered clinical trials in the last quarter of 2009.

Prosensa was founded in 2002 and is located in Leiden, The Netherlands. The company works closely together with Leiden University Medical Center (LUMC) and is supported by a consortium of leading biotech investors, including Abingworth, Life Sciences Partners, GIMV, AGF and MedSciences Capital. In October 2009, Prosensa entered into a strategic alliance with GlaxoSmithKline (GSK) to accelerate and broaden the development of its DMD product candidates.