Major investment has accelerated clinical gene therapy research pioneered at the University of Bristol, which has the potential to radically change the treatment of kidney diseases.
Over two million people worldwide with chronic kidney diseases receive treatment with dialysis or a kidney transplant to stay alive. However this number may only represent 10 per cent of people who need treatment to live.
Kidney diseases have historically been disproportionately expensive to treat. Advances in treatment have lagged significantly behind other diseases such as cancer and heart disease.
Gene therapy is a technique which replaces or alters a faulty gene or adds a new gene to treat or prevent disease, instead of using drugs or surgery.
Moin Saleem, Professor of Paediatric Renal Medicine at Bristol Medical School, University of Bristol, is pioneering gene therapy for kidney diseases. He heads Bristol Renal, a world leading group researching glomerular diseases (diseases which affect the glomerulus – the kidney’s blood filtration unit). His gene therapy programme is focused on targeting podocytes, which are cells in Bowman’s capsule in the kidneys that wrap around capillaries of the glomerulus.
UK healthcare investment company Syncona Ltd. invested £45 million in Purespring Therapeutics, a spin-out company founded by Professor Saleem. This was the largest Series A investment in a new biotech company in 2020.
This is a significant step forward in the innovation of new therapies for kidney diseases. Syncona’s investment has accelerated Purespring’s clinical gene therapy research which directly targets the glomerulus. This could see treatment progress from the lab to patients in a matter of years.
At the time of the investment, Professor Saleem said:
“This is an incredible opportunity to develop transformational treatments for kidney disease. Gene therapy has come of age in certain areas, but a major challenge in complex solid organs is to precisely target the genetic material to the correct cell type. Using accumulated expertise in the Bristol Renal research group we have solved this crucial hurdle, putting us in a position to deliver curative gene therapy to patients with chronic and intractable kidney diseases. Syncona have had the foresight to see this potential, and partnering with their world-leading gene therapy experience is the best possible springboard to successfully bring this technology to patients.”