Business and Product Development Strategy
Prosensa was established to develop novel treatments for life-threatening diseases based on its RNA modulation platform. Prosensa intends to discover, develop and commercialize these products and to create a specialized orphan drug franchise. The company wants to address diseases with a large unmet medical need, for which no curative treatments exist today.
It is Prosensa’s objective to reach and help patients globally. To accelerate its global development and to ensure broad patient access, the company may consider selected partnering agreements. A first example is the strategic alliance which was recently signed with GlaxoSmithKline (GSK). This partnership will facilitate a faster global development of the company’s most advanced Duchenne products, while allowing Prosensa to retain commercial participatory rights. In addition, Prosensa has retained full commercial rights to develop products which target additional subpopulations of Duchenne patients.
Prosensa will concentrate its development and commercialization efforts on the neuromuscular and neurodegenerative space. The company intends to establish itself as a highly specialized neuromuscular franchise, with leading products in Duchenne Muscular Dystrophy and other disorders such as Myotonic Dystrophy and Huntington’s Disease. Its RNA modulating platform however has broader applications in other therapeutic areas. In selected areas, Prosensa will deliver the initial (pre-clinical) proof of concept and try to selectively partner out further development to other parties.