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Bristol secures £45M to advance gene therapy treatment of chronic kidney diseases

Press release issued: 19 November 2020

The University of Bristol has secured a £45million deal to advance its groundbreaking gene therapy technology for chronic kidney diseases. The commitment, made by healthcare company Syncona Ltd to Bristol spin-out Purespring Therapeutics, aims to address a global unmet need for renal conditions in one of the largest single investments made to a new UK university biotech company.

Over two million people worldwide currently receive treatment with dialysis or a kidney transplant to stay alive, yet this number may only represent ten per cent of people who need treatment to live. Until now, advances in the treatment of kidney diseases have lagged significantly behind other diseases such as cancer and heart disease.

This investment marks a significant step forward in the innovation of long overdue new therapies for kidney diseases, which have historically been disproportionately expensive to treat.

Gene therapy — 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, offers a potential new type of treatment for renal conditions.

Syncona’s £45 million investment to Purespring will be used to progress to the clinic gene therapy research pioneered by Professor Moin Saleem, Professor of Paediatric Renal Medicine at Bristol Medical School and Dr Gavin Welsh, Associate Professor of Renal Medicine. Professor Saleem’s work is the only study to date (as yet to be published) to have successfully demonstrated disease rescue in animal models using this technique for a kidney disorder called nephrotic syndrome.

Purespring will develop gene therapies directly targeting the glomerulus in the kidney, which could see treatment progress from lab to patients in three or four years. The company will also have access to an in-vivo functional screening platform, FunSel, to screen for cell-specific protective factors delivered via gene therapy, that could have applications across several kidney diseases. FunSel has been developed by Professor Mauro Giacca at Kings College London.

Professor John Iredale, Pro Vice-Chancellor for Health and Life Sciences at the University of Bristol, said: "Syncona’s expertise in gene therapy and landmark investment in Bristol spin-out Purespring marks an exciting new venture to progress Bristol’s breakthrough discoveries in the treatment of kidney diseases. Purespring’s gene therapy platform has enormous potential to improve outcomes in patients with kidney diseases and is a major leap forward for renal therapeutics globally.”

Professor Moin 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.”

Chris Hollowood, CIO, Syncona Investment Management Limited, said: “Purespring is the sixth gene therapy company to be founded by Syncona and clearly demonstrates our proprietary company creation approach. In Moin and his team, we are collaborating with clinical and scientific leaders and working in target tissue amenable to gene therapy, whilst the collaboration with Mauro provides a path for gene therapy to fulfil its promise in highly prevalent chronic degenerative conditions. We look forward to building a world class company around this innovative science, in order to develop therapies with the potential to deliver dramatic impact for patients. Purespring is an exciting addition to our gene therapy platform, where we are strategically positioned with significant expertise in building fully integrated platform companies.”

Further information

This work was supported by the Elizabeth Blackwell Institute for Health Research, University of Bristol and Medical Research Council.

About Syncona
Syncona (LON: SYNC) is a healthcare company focused on founding, building and funding a portfolio of global leaders in life science. Our purpose is to invest to extend and enhance human life. We do this by founding and building companies to deliver transformational treatments to patients in areas of high unmet need.

Our strategy is to found, build and fund companies around exceptional science to create a dynamic portfolio of 15-20 globally leading healthcare businesses for the benefit of all our stakeholders. We focus on developing treatments for patients by working in close partnership with world-class academic founders and management teams. Our strategic balance sheet underpins our strategy enabling us to take a long-term view as we look to improve the lives of patients with no or few treatment options, build sustainable life science companies and deliver strong risk-adjusted returns to shareholders.

About ICGEB and FunSel
Established in 1983 as a special project of UNIDO, the International Centre for Genetic Engineering and Biotechnology - ICGEB is an independent intergovernmental organisation since 1994 with HQ in Trieste (Italy) and with additional laboratories in New Delhi (India) and Cape Town (South Africa). As of today, it counts 65 Member States and 20 signatory countries. The ICGEB is a not for profit IGO – any revenues generated are re-invested in research and in the funding programmes for capacity building in its Member States. The Vision of the ICGEB is to be the world’s leading intergovernmental Organisation for research, training and technology transfer in the field of Life Sciences and Biotechnology. Its Mission is to combine scientific research with capacity enhancement, thereby promoting sustainable global development (

FunSel is an in-vivo functional screening platform. It was developed at ICGEB by Professor Giacca and his team while he served as the Director-General of the organisation until 2019. He continues to head the Molecular Medicine laboratory at ICGEB Trieste, Italy.

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