(Coordinator: Francesco Muntoni, London, UK): Phase I/IIa clinical trial in Duchenne muscular dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53.

Submitted under: HEALTH.2012.1.4-4. Targeted nucleic acid delivery as an innovative therapeutic prophylactic approach

The idea of the SKIP-NMD project is to perform a safety assessment of a lead PMO compound to skip exon 53, and perform a randomised study looking at safety, biochemical efficacy and exploratory clinical efficacy of this novel antisense in ambulant boys affected by DMD. Exploratory measures will be analyised including muscle MRI and serum biomarkers to assess non invasively response to therapy.

Three UK charities (AD; MDC; DMS support group), one Italian (DMP Parent Project Italy, and one French (AFM) are involved in this project.

There are a number of companies involved in the project, from those involved in PMO production, to companies which manufacture some of the new outcome measure equipment, to companies involved in preclinical toxicology and MRI data acquisition and analysis.

Project length - 3 years
Project award - up to €5.58million
12 Apr 2017