Prosensa
| Company type | Public |
|---|---|
| Nasdaq: RNA | |
| Industry | Biotechnology |
| Founded | 2002 |
| Headquarters | Leiden, Netherlands |
Key people | Hans Schikan (CEO), Berndt Modig (CFO), Giles Champion (CMO), Luc Dochez (CBO) David Mott (Chairman) |
| Products | Pharmaceuticals |
Number of employees | 89 (2014) |
| Website | www.prosensa.com |
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. Prosensa’s lead product, drisapersen has completed Phase III clinical trials. Three additional products, PRO044, PRO045 and PRO053 are in clinical development, and PRO052 and PRO055 are in advanced preclinical development. In parallel, Prosensa has started a discovery research program called PROSPECT, which includes a new and innovative application of its exon-skipping technology platform, applying multiple exon skipping, to specifically target rarer mutations (initially in exon 10 to 30 region) in the dystrophin gene. This approach could have applicability between 5-13% of the DMD population.
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.
Prosensa commenced operations in 2002 and is located in Leiden, The Netherlands. The company works closely together with academia, patient groups and experts worldwide. Prosensa entered into an exclusive licensing agreement in 2003 with Leiden University Medical Center (LUMC) for their proprietary RNA modulation exon-skipping technology to develop treatments for DMD, other neuromuscular disorders and indications outside the field of neuromuscular disorders.