SKIP-NMD

Project Overview

motor nerves

Challenge being addressed

The SKIP-NMD project is designing a drug called an antisense oligonucleotide (AON) to ‘skip’ exon 53. Skipping exon 53 will restore dystrophin production in DMD boys with deletions spanning exons 52, 45-52, 47-52, 48-52, 49-52 and 50-52. Therefore only a subset of DMD boys will be eligible for this treatment. The drug will first undergo pre-clinical tests, followed by a phase I/IIa clinical trial. The project will also develop new outcome measures and biomarkers to ascertain the drug’s effectiveness.

The project follows on from Prof Muntoni’s previous work in the MDEX consortium with Sarepta Therapeutics involving another AON previously shown to safely restore dystrophin production in another subset of DMD boys, by ‘skipping’ exon 51 (Kinali et al, 2009; Cirak et al, 2011). However, different AON are required to skip different exons and so ultimately a panel of such AON drugs is required to ensure as many DMD boys as possible can be treated.

Objectives

The objectives of the SKIP-NMD grant are:

  • design an antisense oligonucleotide drug to skip exon 53 which efficiently restores dystrophin production
  • assess the drug’s safety and efficacy
  • perform a clinical trial in eligible DMD boys
  • develop outcome measures and non-invasive biomarkers to monitor the drug’s success.

Key figures about the Project

  • Start date: 1st November 2012
  • Duration: 36 months
  • EC contribution: €5,512,424.07

The partners include 4 industrial companies and 4 clinical trial centres, across 5 countries (UK, France, Belgium, Italy and USA). More details on the individual partners can be found here.

We will also be working closely with six DMD parent organisations, 3 from the UK (MDC, AD, DFSG), 2 from France (AFM, DPP France) and 1 from Italy (DPP Onlus). These parent advocacy groups will contribute to general study oversight, clinical trial design and contribute to disseminating SKIP-NMD’s progress to DMD patients and families.