Prosensa is a biotechnology company engaged in 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 and Huntington's disease.
Prosensa's portfolio of clinical and pre-clinical RNA-based drug candidates is currently focused on the treatment of DMD. The lead product, drisapersen, is being developed in collaboration with GlaxoSmithKline and is currently in Phase III clinical trials. Three additional products, PRO044, PRO045 and PRO053 are in clinical trials, and PRO052 and PRO055 are in advanced preclinical development. In total, Prosensa's preclinical and clinical compounds have the potential to treat approximately 40% of DMD patients. In parallel, Prosensa has started a research program called PROSPECT, which includes a new and innovative application of its exon-skipping technology platform to specifically target rarer mutations in the dystrophin gene.
In addition to its clinical trials, Prosensa is running a Natural History Study, which involves trial sites in 10 countries and is currently recruiting. The purpose of this study is to characterize the natural history and progression of DMD, to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
Details of the clinical trials, such as inclusion/exclusion criteria and trial sites, are posted on the website www.clinicaltrials.gov and all required approvals of authorities and ethics committees are granted.
Prosensa commenced operations 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.
"We aim to develop innovative, RNA-based therapeutics to address unmet medical needs for patients with genetic disorders."
- Founded in 2002
- Located in Leiden, The Netherlands
- ± 85 employees
- RNA modulation technology platform
- Focus on NeuroMuscular Disorders
- Clinical portfolio in Duchenne Muscular Dystrophy