The main goal of the FP7 funded SCOPE-DMD project is to further advance and accelerate the development of PRO045, an exon-skipping compound for DMD. PRO045 is currently in a Phase IIb dose-escalating clinical trial to assess its safety and efficacy. The chemically-modified AON induces exon 45 skipping in the dystrophin gene and could be suitable for approximately 8% of all DMD patients.
Funder: EU FP7-HEALTH-2013-INNOVATION-2
Grant value : € 6m
Project length - 3yrs