Help the Heart

We will fund ambitious, translational or clinical projects which address the following areas:

• Understanding the unique features of the DMD heart and cardiac disease progression in DMD.
• Innovative treatment strategies and approaches (e.g. who & when to treat- young patient, early disease stage versus older patients, advanced disease stage), in the context of the latest clinical recommendations and cardiac standard of care for DMD.
• How can we evaluate the cardiac response to therapies better?
• Characterisation of heart phenotypes in DMD to help understand the efficacy and safety profile of disease modifying treatments.

Examples of projects we could support:

• Biomarker development and validation for cardiac disease monitoring (e.g. blood biomarkers, imaging biomarkers). To speed up project delivery, we will prioritise proposals with evidence of access to a pre-consented patient cohorts or samples.
• Clinical drug repurposing programme looking at therapies, as standalone or combinations, that could be repositioned for DMD.
• Development of translational and clinically predictive tools to screen and characterise the safety and efficacy profile of drugs to treat the DMD heart.

Under this call, we will aim, where possible, to fund projects that can be complimentary and synergise with each other, and work with applicants to join them up to ensure the biggest impact.

Therapeutic projects expressions of interest may be triaged to the Strand Two – Therapeutic grant call, which will launch later this year, if deemed appropriate by the Duchenne UK team.

Our funding is open to universities, research institutes and small companies (micro and medium) worldwide. Projects are expected to last up to two years, with total project costs up to £500,000.

Higher project costs may be considered subject to how many projects will be funded. Matched funding from other funders will be considered for projects with costs above £500,000.

Within the calls scope described above, we will fund projects that meet the following criteria:

• Ready to enter the clinic or aimed at reaching the clinic within 2-3 years from funding.
• For drug projects, based on a strong Target Product Profile, which clearly demonstrates the rationale for superiority over current SoC therapies and novel therapies in clinical development.
• Have reached robust proof of mechanism as relevant (e.g. biological evidence of an effect which is DMD relevant/specific).
• For drug projects, have preliminary evidence of efficacy or relevance in DMD relevant preclinical models.
• Earlier stage proposals may be considered if they are highly clinically relevant, innovative and disruptive, and our funding is uniquely positioned to enable progress.

We will NOT fund:

• Projects that are very early stage and have no preliminary mechanistic evidence of the proposed drug therapy having an effect in the target tissue in DMD.
• Technology development projects, including the development of preclinical model (in vivo and in vitro).

• Projects will be accepted from when the call is announced and throughout the year. We may decide to close the call earlier subject to level of engagement in the research community and number of proposals received.
• For proposals submitted under this call we will use our standard process for Response Mode Funding.
• Expression of interest (EOI) proposals or enquires can be submitted to [email protected] referencing Cardiac Theme Call EOI in the subject of the email.
• If deemed appropriate, we will invite you to submit a full proposal using our standard template.