SKIP-NMD

Work Packages

EU grants are divided up into work packages (WPs). Each WP describes a particular part of the project and led by one of the partners. WP can run con-currently or sequentially. SKIP-NMD has 8 WPs as follows:

  • WP1- Choice of SRP-4053 RNA sequence.

To establish the RNA sequence needed for optimal skipping of exon 53. Tests will be performed in the laboratory on DMD patient muscle cell lines using a number of different sequences to determine this. The selected lead sequence will be called SRP-4053.

  • WP2- Pre-clinical testing of SRP-4053.

Sarepta Therapeutics will synthesize SRP-4053 and pass to partner Charles River Laboratories for the pre-clinical toxicology studies required by the regulatory authorities. This is to ensure SRP-4053 is safe.

  • WP3- Clinical trial design and regulatory authority submission.

The clinical trial design, study protocol and ethical application will be finalized resulting in a standard operative procedure manual suitable for submission to the regulatory authority to gain clinical trial consent.

  • WP4- Clinical trial patient recruitment.

Patients suitable for exon 53 skipping will be recruited and the clinical trial conducted across the 4 clinical trial centers.

  • WP5- A natural history study of non-invasive biomarkers in DMD.

This will run in parallel with WP5.

  • WP6- Ethics and Parent organizations forum.

Through interacting with the parent advocacy groups, this WP will ensure correct ethical approaches are used both in the clinical trial and natural history studies.

  • WP7- Dissemination to the academic and parent organizations.

SKIP-NMD’s progress will be disseminated through a variety of means e.g. SKIP-NMD and parent advocacy web sites, scientific meetings and journals.

  • WP8- Project management and administration.

SKIP-NMD reports to the EC through University College’s European Research and Development Office (ERDO) to ensure efficient organization and management of the project.

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