New therapy offers hope for children with genetic kidney disease

13 August 2026

In new results, published in Science Translational Medicine, Dr Saif Malik, Dr Jennifer Chandler and Professor David Long at University College London (UCL) Great Ormond Street Institute of Child Health, with funding from Kidney Research UK, have developed a new targeted therapy that could slow the progression of genetic childhood glomerular disease.

A childhood kidney disease with no existing treatments

Glomerular disease is a condition that damages the tiny filtering units inside the kidneys, known as the glomeruli. These filters normally remove waste from the blood while keeping important proteins in the body.

But in children with kidney disease, genetic changes prevent the glomeruli from working properly. Over time, proteins leak into the urine, kidney cells are damaged, and scar tissue builds up.

As the disease progresses, many children develop kidney failure and may need lifelong dialysis or a kidney transplant. Now, researchers have designed a precision RNA therapy that aims to protect these vulnerable kidney filters and tackle the underlying cause of the disease.

three researchers in the lab, all three wearing white lab coats — Dr Jennifer Chandler, Dr Saif Malik and Professor David Long

A precision approach to protecting the kidney

Rather than correcting a specific genetic mutation, the new therapy targets a biological pathway involved in the development of kidney damage.

Professor David Long in the research lab — Professor David Long

RNA carries the instructions cells use to make proteins. The treatment delivers RNA that restores levels of angiopoietin-1, a naturally occurring protein that helps maintain healthy blood vessels and supports the kidney's filtering function. Reduced levels of angiopoietin-1 have been linked to this genetic childhood disease, but the same pathway is also involved in more common forms of kidney disease in adults.

Professor David Long said: “For families affected by childhood kidney disease, the lack of treatments can be devastating. By targeting the pathway that drives kidney damage, this approach could open the door to new therapies that protect kidney function — not only for children with rare genetic conditions, but potentially for many more patients with kidney disease.”

Dr Saif Malik added: “This research takes our understanding of angiopoietin-1 from the laboratory towards a potential treatment for a disease where children currently have no options to slow its progression.”

Delivering RNA directly to the kidney

RNA therapies have already been successfully used in other areas of medicine. However, this technology has not previously been adapted to directly deliver treatments to the kidney.

To overcome this challenge and safely deliver the RNA, Saif, Jennie and the team packaged the RNA inside tiny particles called lipid nanocomplexes. They also developed a minimally invasive, ultrasound-guided technique to deliver the treatment directly into the kidney's main blood vessel – known as the renal artery. The nanocomplexes were designed to target the kidney cells most affected by the disease, improving delivery to the glomerulus while limiting exposure elsewhere in the body.

When tested in laboratory models, the targeted therapy reduced protein leakage into the urine, preserved the specialised cells responsible for filtration and reduced scarring within the glomeruli.

“With funding from Kidney Research UK, we have been able to develop and advance this targeted RNA approach, showing its potential to protect the kidney’s filtering units and tackle the underlying drivers of disease. For a condition where no treatments currently exist to slow progression, these findings represent an important step towards developing disease-modifying therapies that could change the future for patients.” Dr Jennie Chandler.

A platform for future kidney therapies

For children and families affected by rare genetic kidney diseases, this research offers hope for the first treatments that could slow disease progression. But the potential impact may reach much further: because the therapy targets a pathway involved in many forms of kidney damage, it could one day help patients with a broader range of kidney conditions, including adults with more common diseases such as diabetes.

There is still important work ahead before the treatment can reach patients. Researchers will need to carry out further safety studies and develop ways to produce the therapy at larger scale. However, the findings mark an important step towards a future where kidney disease could be treated by protecting the kidney itself.

David Crosby, Chief research officer at Kidney Research UK said: “Inherited, rare and paediatric kidney diseases have a huge health, emotional and economic impact but they lag behind other conditions when it comes to treatment development, trials and breakthroughs – which is why it’s so crucial to see charities and academic institutions backing innovative research like this.

“This pioneering technique from UCL GOS ICH. We are proud to support the team behind this important research. With our funding, they have taken an innovative approach that combines targeted delivery with precision RNA therapy — a strategy that could transform how we treat glomerular disease. It offers real hope of changing the outlook for children with genetic glomerular disease and could potentially lead to even wider use for the benefit of thousands of patients at risk of kidney failure.”

This research also received funding from Great Ormond Street Hospital Charity (GOSH Charity) in partnership with Life Arc.