World Kidney Day 2023: Transforming Kidney Disease through Podocyte Gene Therapy
Authors: Pille Harrison, Moin Saleem & Aoife Waters
9 March 2023
‘There is no cure’ is a hard fact that newly diagnosed patients with chronic kidney disease (CKD) often hear.
It is estimated that more than 850 million people suffer from CKD globally1: that’s over 10% of the world’s total population facing a future without a long-term cure. Purespring is hoping to transform the course of CKD by the application of kidney-directed gene therapy, and we intend to do this by targeting a specialised kidney cell: the podocyte. The origin of harnessing podocytes to treat CKD by gene therapy comes from pioneering work lead by Purespring co-founder and Professor of Paediatric Renal Medicine at Bristol University, Moin Saleem.
While there are known risk factors, including other health conditions and lifestyle choices (e.g., smoking), CKD due to genetic mutations occurs as frequently as 1 in 1,000 births. Current treatments, at best, manage or modify symptoms, they don’t treat the underlying causes of disease. Patients progress to dialysis and, eventually, kidney transplantation, with a lifetime requirement of strict adherence to their medications. Even then, patients can require more than one transplantation during their lifetime.
Early intervention with an effective transformational therapy, such as gene therapy, which can protect or modify damaged or dying podocytes from a single administration in a lasting way, could avoid the need for transplantation completely. A real opportunity exists for the healthcare industry to find new long-term solutions for CKD patients, their families, and carers.
The kidney has a sophisticated filtration system which is tightly controlled by podocytes, specialised cells found in the glomerulus, at the centre of the kidney’s filtering units. Diseases affecting the glomerulus are increasing and leading to an increase in CKD, making it a key target for more effective treatments. Protecting podocyte health, therefore, is a crucial strategy for developing treatments for CKD.
Podocyte-directed gene therapy offers exciting new prospects for durable treatment benefit over existing strategies.
- Nephrol Dial Transplant (2019) 34: 1803–1805