GOSH Charity commits largest amount yet towards 2023/24 National Funding Call

This year’s funded projects aim to tackle some of the most rare and complex childhood illnesses and cover a range of therapy areas.

• Five projects are looking to gain a better understanding of disease or improve the diagnosis or treatment options for a particular disease. For example, overcoming drug resistance in acute lymphoblastic leukaemia, improving diagnosis for a rare form of epilepsy and methods of preventing and treating a rare neural tube defect similar to spina bifida.
• Seven projects are focussed around developing novel gene therapies that target the specific disease – for example, accelerating gene therapy for an incurable muscle disease called X-linked myotubular myopathy (XLMTM), developing gene therapy for Norrie disease and using gene therapy to treat a severe form of epilepsy.

The successful applicants are based at institutions across the UK, including the University of Edinburgh, University of Southampton, Keele University, Newcastle University and University College London.

Professor Brian Bigger, Professor of Advanced Therapeutics at the University of Edinburgh, says: “I am delighted that my research is one of the 12 projects to have been awarded funding from GOSH Charity’s National Call this year.

“Hunter syndrome and Sanfilippo syndrome are progressive diseases that have an immeasurable impact on both the child and their family. This investment from the National Call will mean that my team and I can move a step closer in developing a treatment for the hundreds of families across the UK and beyond facing the devastating consequences of these conditions.”

Excitingly, this year, for the first time, the charity established a Patient Benefit Panel to contribute to the assessment of projects.

The Panel consists of five members – each of whom have lived experience of care in a paediatric healthcare setting, either as a patient themselves, a parent or loved one.

The group helped to shape the research that the charity funds, by ensuring patients and their families are appropriately considered, engaged, and consulted in the research and that the research can be communicated to non-expert audiences.

As part of our 2023-2028 research strategy and our ambition to transform the lives of seriously ill children through research-led care, we are making £2.5 million available for the 2024/25 round of the National Call.

Building on the success of previous rounds, we will continue to partner with other charities to jointly award funding for exceptional projects in specific disease areas.

This year we are proud to partner with The Norrie Disease Foundation, Myotubular Trust, Cure DHDDS and Dravet Syndrome UK.

Are you a researcher? The 2024/25 GOSH Charity National Funding Call is currently open for applications and the submission deadline is 3pm on Wednesday 26 June.