fbpx

Therapeutic grant call

Accelerating research for transformative therapies for Duchenne muscular dystrophy (DMD) - expression of interest proposals invited

Developing transformative DMD therapies

Despite the progress in the development of treatments addressing the root cause of DMD through delivery of replacement (micro) dystrophin (i.e. gene therapy), we are still not able to achieve transformative change in the course of the disease and its devastating impact on the lives of people living with DMD.

We face major challenges as we seek to achieve marked improvements in clinical benefit, including: 

– Limited clinical efficacy and benefit profiles of current dystrophin replacement approaches available in the clinic

– DMD genetic profiles associated with immunogenicity and significant concerns on safety, which will require immunomodulation/immunosuppression and more sophisticated screening strategies

– Sub-optimal, viral based, drug-delivery systems used for genetic therapies which: (1) preclude all patients to have access to genetic treatments due to the host pre-existing antibodies to the virus (2) prevent re-dosing, leading to issue of effect durability, due to the host response to viral vectors (3) can only deliver small genetic sequences, leading to limited functional effects associated with the shorter protein (instead of a fully functional, full-length protein)

– Lack of clear understanding of the impact of these treatments on the heart (efficacy and safety)

– Lack of understanding of the long-term clinical benefit (and safety) of dystrophin replacing therapies, which require a better mechanistic understanding of how these drugs work at a cellular and muscular tissue level

Supporting research to accelerate progress

We want to accelerate progress of novel therapeutic programmes that address these challenges to ensure new, safer, and more effective treatments reach the clinic as soon as possible or within up to three years from us funding the programme.

Scope 

We are seeking to fund ambitious, translational projects driving the next generation of transformative DMD therapies, which have the potential to disrupt the current therapeutic landscape leading to marked improvements in the safety, efficacy, including cardiac outcomes, and delivery profile, over current Standards of Care therapies and therapies in the clinical development pipeline.

Please find the full description of the funding call’s scope here.

Eligibility

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.

Submit your expression of interest proposal by the 23:59 on 4 August – see below for more details.

Submission and review process

Here is an overview of the submission and review process.

If you would like to discuss this funding call with a member of the team or have any questions, you can email us at [email protected].