Children’s charity Action Medical Research and independent medical research charity LifeArc are pleased to announce a fourth joint funding round aimed at supporting UK-based translational research projects that could lead to new interventions (therapeutics, diagnostics and devices) for infants, children or young people with a rare disease.

The charities have created a £1m fund for researchers seeking additional funding to progress promising translational research projects aimed at addressing a clear unmet medical need for children and young people with a rare disease.

The joint call is open to projects developing a broad range of intervention types (therapeutics, diagnostics, and devices), including the development of targeted gene therapy approaches that could be particularly well-placed to benefit the high proportion of monogenic paediatric rare diseases. Further information on remit, eligibility and how to apply can be found on the Action Medical Research website.

This is the fourth joint funding call issued by the charities. Projects successfully funded in the last round are expected to be announced this summer. In the 2020 funding round three projects were jointly funded; one optimising new drug treatments for Duchenne muscular dystrophy, another aiming to improve diagnosis and monitoring of hypertrophic cardiomyopathy in children, and a third project looking to develop new drug treatments for pyridoxine dependent epilepsy.

Two projects in the 2019 funding round were funded; one assessing whether the combination of existing drugs could be used as a new treatment for childhood leukaemia and another aiming to improve the effectiveness of a stem cell gene therapy for Hunter Syndrome.

A recent report for 2022 Rare Disease day focused on the impact an improved stem cell gene therapy could have for children with Hunter syndrome, highlighting how research funded by this joint scheme could have real benefit for patients.

Dr Catriona Crombie, Head of LifeArc’s Philanthropic Fund said: “Through this joint funding call, LifeArc and Action Medical Research have already committed over £1.1m to support five translational research projects focused on developing improved therapies and diagnostics for children and young people with rare disease.

“Our continued partnership with Action Medical Research will provide the funds and expertise to help translate further high-quality research into interventions that could benefit paediatric patients with rare disease.”

Dr Tracy Swinfield, Research Director at Action Medical Research said: “Our ongoing partnership with LifeArc enables us to fund cutting-edge medical research that could have a significant impact on the treatment of babies, children and young people living with rare and sometimes devastating disease. ”

The deadline for submitting an outline application is 21 June 2022. Please click here for more information on the joint fund.

– Ends –

Notes to editors

How to apply

Outline applications can be made via the Action Medical Research website and will need to meet the guidelines provided. Projects of up to three years’ duration and costing a maximum of £250,000 will be considered. Researchers invited to submit a full application are expected to be informed of a final funding decision in summer 2023.

For more information on Action Medical Research

AMR LogoGrace Wardley
Communications and Engagement Officer
+44(0)7725 090 008

Follow Action Medical Research

Twitter: @actionmedres and @amr_events 
Instagram: @actionmedres and @actionevents

Action Medical Research is a leading UK-wide charity saving and changing children’s lives through medical research. For 70 years we’ve helped pioneer ways to prevent disease and develop treatments benefiting millions of people. Our research has helped to beat polio in the UK, develop ultrasound in pregnancy, fight meningitis and prevent stillbirths. But we urgently need to develop more new treatments and cures for sick babies and children and we can’t do it without you.

Join our fight for little lives today.

Charity reg. nos 208701 and SC039284

For more information on LifeArc

Surinder Maan
Communications Manager
+44 (0)20 7391 2754

Follow LifeArc on LinkedIn or Twitter

LifeArc is a self-funded medical research charity. Our mission is to advance translation of early science into health care treatments or diagnostics that can be taken through to full development and made available to patients. We have been doing this for more than 25 years and our work has resulted in a diagnostic for antibiotic resistance and four licensed medicines. Our success allows us to explore new approaches to stimulate and fund translation.

LifeArc is proud to be renewing joint funding with Action Medical Research, which is the latest in a series of strategic collaborations that leverage LifeArc’ s expertise in translational science – advancing strong discoveries from the lab to a point where rare disease patients can benefit. This includes a recent £8m commitment made by LifeArc to support the delivery of new gene therapy treatment through a unique partnership with the MRC.

Through this partnership, LifeArc is partly funding a network of cutting-edge facilities – also known as Gene Therapy Innovation Hubs – to enable the progress of novel, academic gene therapy research into early-stage clinical trials and bring a new generation of medicines to healthcare. It is hoped that promising gene therapy approaches, such as those supported by LifeArc’s joint fund with Action Medical Research, may benefit from interaction with the Gene Therapy Innovation Hubs as they progress towards clinical trials.