Investing in the first step of research for rare and complex childhood diseases
25 Aug 2026, 12:25 p.m.
We’re proud to be funding six new research projects. Each one is taking the first step for a new childhood disease research question.
Great Ormond Street Hospital Charity (GOSH Charity) has awarded over £470,000 in funding through our Lift Off Grant: Discovery scheme.
Funding six new research projects
The funded projects are all led by staff at Great Ormond Street Hospital (GOSH) and its research partner, the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH). Project leads hold a variety of roles, including Clinical Lecturer, PhD student, and Professor.
Supporting early career researchers
We’re delighted that three of the six successful applicants are early career researchers.
We encourage early career researchers to apply for our funding as we want to maintain and increase the number of brilliant people working in rare paediatric disease. This supports the ambition of our research strategy to transform the lives of children with rare and complex conditions through research-led care.
Personalising early warning scores for children with rare and complex conditions
One of the early career researchers to receive funding is Dr Alexander Brown, a Clinical Lecturer in Paediatric Intensive Care at GOSH. Dr Brown’s project aims to improve the way that hospitals identify when children with complex or rare health conditions are becoming seriously unwell.
Current early warning scores are based on what is considered ‘normal’ for healthy children and may not work as well for children with rare and complex conditions, such as those seen at GOSH. Such conditions can affect a child’s usual heart rate, breathing or other vital signs.
In this project, Dr Brown will use anonymised data to explore ways of improving early warning scores.
By using advanced data analysis to personalise these scores and identify important changes over time, Dr Brown hopes to help healthcare teams recognise deterioration earlier and improve patient safety.
“This funding will allow us to use cutting-edge data science to improve the systems we use to monitor children in hospital for signs of deterioration. By taking into account each child’s individual circumstances and underlying health conditions, we aim to give clinicians more accurate information, earlier, so they can act when it matters most.”
Developing a new way to deliver gene therapies to children with cystic fibrosis and other serious lung conditions
Another successful applicant was Professor Steven Hart, Professor in Molecular Genetics at UCL Great Ormond Street Institute of Child Health. Professor Hart’s project aims to develop a new way of delivering gene therapies to children with cystic fibrosis and other serious respiratory conditions.
Children with cystic have thick, sticky mucus in their lungs. This mucus makes it difficult for the lungs to clear infections and causes long-term damage over time. Gene therapies have the potential to treat the underlying genetic cause of these conditions. However, most gene therapies are designed to be inhaled into the lungs, and the thick mucus acts as a barrier that prevents these treatments from reaching the cells they are intended to help. As a result, promising therapies fail in clinical trials because they cannot penetrate the mucus barrier by inhalation.
In this project, Professor Hart will develop particles that can carry gene therapies through the bloodstream and into the lungs where they are needed.
This research could provide an entirely new way of delivering gene therapies to the lungs of children with cystic fibrosis and other serious respiratory diseases. By bypassing the mucus barrier, it may allow treatments to reach cells that are currently inaccessible using inhaled medicines.
“We are incredibly grateful to GOSH Charity for supporting this project. This funding will allow us to test a completely new way of delivering genetic medicines to the lungs of children with serious respiratory diseases such as cystic fibrosis. While this research is still at an early stage, it could open the door to new treatment approaches for children who currently have very limited options, including those with rare genetic forms of disease that cannot be treated with existing medicines.”
Apply for funding
Lift Off Grants: Discovery are one of our research funding schemes. Please visit our research funding pages to explore all opportunities.
If you'd like to sign up to our GOSH Charity research newsletter to hear when funding schemes open and other news, please email researchcomms@gosh.org.