We’re proud to have funded new research with Great Ormond Street Hospital Charity (GOSH Charity) and Kidney Research UK that marks an important step towards developing a new treatment for serious and life-threatening genetic kidney diseases affecting children.

New research, published in Science Translational Medicine and pioneered by a team from UCL Great Ormond Street Institute of Child Health (UCL GOS ICH), is giving new hope to seriously ill children facing genetic glomerular disease, a severe, life-limiting kidney condition which currently has no treatment.

Affecting children from a very young age, genetic glomerular disease prevents the kidneys from filtering waste and excess fluid from the blood properly. There are currently no treatments that can stop the condition from worsening, leaving many children with kidney failure before their third birthday and needing long-term dialysis, and eventually a kidney transplant. The disease affects tiny structures inside the kidney called glomeruli. These microscopic filters clean the blood, keeping important proteins in the body while removing waste.

Although childhood glomerular disease is rare, collectively glomerular diseases in children and adults make a significant contribution to the £6.4 billion the NHS spends each year managing chronic kidney disease.  The latest figures from April 2025 to April 2026 show that there are now 138 children waiting for a kidney transplant in the UK.

Research led by Dr Jennie Chandler, Professor David Long and Dr Saif Malik from the UCL GOS ICH, has pioneered a new treatment that gives cells new genetic instructions, known as RNA therapy, that could help restore an important protein, improving the kidney’s ability to filter properly and support healthy kidney function.

As part of the study we helped fund, the team also demonstrated a new way of delivering the treatment directly to the kidney, meaning more of the therapy can reach the damaged kidney tissue, while reducing side effects elsewhere in the body.

Professor David Long, Professor in Paediatric Nephrology and Head of the Developmental Biology and Cancer Department at the UCL GOS ICH, said:

“Funding by GOSH Charity and LifeArc has meant we have been able to explore and advance new generations of treatments designed to delay kidney disease progression and change the way patients are cared for. This research has the potential to be transformative, not just by improving kidney function but by removing significant physical and emotions burdens that children and their families carry when facing this condition.

In addition to that, the approach of delivering RNA medicines directly to the kidney’s filters could potentially be adapted to treat other diseases in the future, opening new possibilities for developing more targeted treatments, offering hope for patients with conditions that are currently difficult to treat.”

Although the treatment is still in the very early stages of development, the researchers hope that with the necessary funding and regulatory approvals, they can take the next steps towards evaluating its potential in patients within 3 to 5 years. The findings offer hope that, one day, children with genetic kidney disease could have their kidney function preserved for longer, delaying the onset of kidney failure and potentially reducing the need for long-term dialysis or kidney transplant.

Sam Barrell, CEO of LifeArc, said: “Rare disease can place an enormous burden on children and families, often from the earliest years of life. Too many families still face the uncertainty of limited treatment options which is why research like this matters: it offers hope that we can begin to change the course of serious conditions, rather than simply managing the damage they cause.

We’re proud to have supported this work through our partnership with GOSH Charity. It is exactly the kind of pivotal research we need to back: science with a clear clinical purpose and a route towards treatments that could one day give children the childhood they deserve.”

By slowing disease progression, future treatments could help children spend more time at home, in school, and doing the things they love, while spending less time undergoing intensive medical treatments. This could give seriously ill children the opportunity to experience more of the moments that make childhood special.