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) and Huntington's Disease (HD).

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, drisapersen (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/IIa study. An open label extension study of this Phase I/IIa study is on-going and the 12-week data were published in NEJM in April 2011. A phase III study started early 2011 and has recruited all patients. PRO044, the company’s second product candidate, which addresses another subpopulation of Duchenne patients, currently is in a Phase I/IIa study. It is anticipated that another two additional compounds, PRO045 and PRO053, will enter the clinic in the first half of 2013.

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.

Mission Statement