£1 Million Fund to Support Translational Muscular Dystrophy Research

Muscular Dystrophy UK and LifeArc have launched a new fund to support translational research projects to accelerate the development of new therapies for Congenital Muscular Dystrophies.

Muscular Dystrophy UK (MDUK) and LifeArc are co-funding the Muscular Dystrophy Translational Research Fund to support projects with the potential to accelerate new treatments for Congenital Muscular Dystrophies (CMD).

CMDs are typically detected at birth or in the first few months of life and share a common muscle pathology. There are no current effective treatments for CMDs, which are caused by mutations in different genes and vary in severity of symptoms.

MDUK and LifeArc are seeking to encourage and support treatment-focused, milestone-driven medical research projects, which are already on the translational pathway, to develop effective, new treatments for CMD. Funding in the order of £300,000-£400,000 will be available to support projects over a maximum duration of three years.

Projects must be led by research-active academics or clinicians based at UK institutions. The project lead should have relevant expertise and experience and be actively engaged with the project. Collaboration with industry and other academic partners in the UK and overseas is encouraged, where relevant.

Proposed projects should:

  • Seek to develop a therapeutic intervention to address the need for more effective treatments for CMD
  • Have a strong scientific rationale
  • Be a target-driven, with milestones and a credible delivery plan
  • Have a clearly set-out route to CMD patients.

Projects/activities eligible for support include:

  • Developing candidate therapeutic entities (eg drug discovery)
  • Pre-clinical testing of novel therapeutic entities in vitro or in vivo
  • Pre-clinical validation for existing therapeutics repurposed for CMD
  • Early-phase clinical trials (phase 1/2) of novel therapeutic entities
  • Early-phase clinical trials of existing therapeutics repurposed for CMD.

Methods of treatment within scope include small molecules, peptides, antibodies, gene therapies (including antisense oligonucleotides), cellular therapies, and regenerative medicine.

It is hoped that projects will generate valuable outcomes that can secure follow-on funding (ie additional translational research funding or partnering with/licensing to industry) to support continued development and progression towards clinical trials.

There is a two stage application process: an Expression of Interest followed by a full application for selected applicants.

Expressions of Interest should be submitted via the LifeArc website by the deadline on 31 May 2022.

(This report was the subject of a RESEARCHconnect Newsflash.)