Novel Therapy for Polycystic Kidney Disease (PKD)

Available for: Exclusive/Non-Exclusive Licensing


Polycystic kidney disease is the most common inherited cause of kidney failure, affecting 1 in 1000 adults and children worldwide. It is characterized by the growth of multiple cysts leading to loss of normal kidney structure and functions and often results in end-stage renal disease. The researchers at UCL Institute of Child Health have demonstrated that treatment with VEGF-C improved disease severity in animal models of PKD.

The Technology and its Advantages

The normal kidney structure is destroyed in PKD by multiple fluid-filled cysts, leading to kidney failure. Currently most treatment strategies target disrupted cellular functions within the cysts themselves but this approach has yet to generate clinically approved therapies for PKD. UCL Institute of Child Health researchers have shown that the microvasculature surrounding kidney cysts shifts from blood to lymphatic endothelial phenotype in PKD. Moreover, their data supports the hypothesis that these blood-to-lymphatic changes are required for cyst growth. Further experiments showed that treatment with VEGF-C, the key regulator of lymphatic growth, survival and migration resulted in significant improvements in disease severity of both, recessively and dominantly inherited polycystic kidney disease. The researchers are currently exploring the delivery to the cyst vasculature using recombinant VEGF and gene therapy.

Market Opportunity

PKD is an Orphan indication. The recessively inherited form of PKD (ARPKD) occurs in 1 in 20,000 and affects mostly children. It can present at any stage of development of a child up to adolescence and most patients need dialysis and kidney transplant due to renal failure. The dominantly inherited form of PKD (ADPKD) is more common, occurring in 1 in 600 and causes kidney failure in half of the cases, usually around middle age. ADPKD sufferers account for 2-3% of all adult dialysis patients. To-date most treatment strategies have targeted disrupted cellular functions within the cysts but this approach has not yet been clinically proven. We are now looking for a partner to develop the findings of UCL scientists into a new therapy for PKD.

Intellectual Property Status

Patent pending (WO214125291)

Further Information

Please contact Dr Chris Baker, Senior Business Manager | T:+44 (0)20 7679 9000 | E:

The technology referred to herein is experimental in nature and UCL Business PLC makes no representations and gives no warranties of any kind, either express or implied, in relation to the technology and, in particular but without limiting the foregoing, UCL Business PLC gives no express or implied warranties of merchantability, satisfactory quality or fitness for a particular purpose.