Translational research into children’s rare diseases enters 5th year of collaborative funding

LifeArc is proud to be renewing our joint funding collaboration with Action Medical Research, a leading UK charity dedicated to saving children’s lives through the application of cutting-edge medical research.

As in previous years, the joint fund will support translational, biomedical research projects from across the UK to develop new interventions (therapeutics, diagnostics and devices) for children and young people with rare diseases. This promises to accelerate existing research that will have a significant impact on the treatment of infants, children, and young people with any of the roughly 7,000 heterogeneous conditions collectively known as ‘rare diseases’.

The joint call is open to a variety of research activities involving:

• The development of candidate therapeutic entities (such as drug discovery)
• Pre-clinical testing of novel therapeutic entities in vitro or in vivo
• Pre-clinical validation for repurposed therapeutics
• Early-phase clinical trials of novel therapeutics or existing therapeutics repurposed for new indications
• Development and testing of diagnostics in pre-clinical or early-phase clinical research settings
• Development and testing of novel medical devices

Projects are expected to address a significant, unmet need for children and young people with rare diseases, have a strong scientific rationale, be target-driven and demonstrate an appropriate strategy for managing intellectual property (IP), to facilitate future development or commercialisation efforts.

A total of £1 million is available to support a small number of ambitious, high-quality projects that could last for up to three years. There will be a maximum of £250,000 allocated to each project.

With details of the 2022 funding round due to be announced later this year, previous funding calls have launched projects investigating:

• Improving a gene therapy for Hunter Syndrome and investigating new combination drug treatments for childhood leukaemia
• New treatments for Duchenne muscular dystrophy, improving the diagnosis and monitoring of hypertrophic cardiomyopathy, and developing small molecule inhibitors for a rare childhood seizure disorder, pyridoxine-dependent epilepsy

The deadline for submission of an outline application is 14 June 2023. Applicants can expect to hear the outcome of their outline applications in October 2023, while the deadline for full applications will likely be in November 2023.

Any additional information required regarding remit, eligibility, and how to apply can be found on the Action Medical Research website.

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