Prosensa Holding B.V. develops therapeutics on the basis of RNA modulation, thereby intervening in an intermediate step in protein biosynthesis.
The company’s lead program is in Duchenne Muscular Dystrophy (DMD), a genetic disorder that blocks the synthesis of an important protein. DMD leads to progressive paralysis and eventually to death. No treatment exists today.
Portfolio: MedSciences Capital B.V. and MedSciences Capital II B.V.
Current stage: NDA submitted. Acquired by Biomarin.