Business and Product Development Strategy
Prosensa was established to develop novel treatments for life-threatening diseases based on its RNA modulation platform. Prosensa is engaged in the discovery, development and commercialization of these products and aims to create a specialized orphan drug franchise. The company strives to address genetic diseases with a large unmet medical need, for which no treatments exist today.
Prosensa's prime objective is to reach and help patients globally. In order to accelerate its global development and ensure broad patient access, the company may consider selected partnering agreements.
In 2009, Prosensa entered into an exclusive worldwide collaboration with GlaxoSmithKline (GSK). This partnership has facilitated faster global development of the company's most advanced Duchenne Muscular Dystrophy (DMD) therapeutics, while allowing Prosensa to retain commercial participatory rights. Prosensa has retained full commercial rights to develop other products which target additional subpopulations of DMD patients.
Prosensa is concentrating its discovery and development efforts on neuromuscular and neurodegenerative disorders. The company intends to establish itself as a highly specialized orphan drug franchise, with leading products in Duchenne Muscular Dystrophy and other disorders such as Myotonic Dystrophy and Huntington's disease. Furthermore, its RNA-modulating platform has broader applications in other therapeutic areas. In selected areas, Prosensa will deliver the initial (pre-clinical) proof of concept and seek partnerships for further development of the product candidates.