October: Purespring Therapeutics | News and features

Purespring Therapeutics became the first company to successfully treat kidney disease models by directly targeting the podocyte – a specialized cell implicated in approximately 60% of kidney disease.

The company has developed a proprietary adeno-associated viral (AAV) gene therapy platform based on pioneering research from Professor Moin SaleemProfessor of Pediatric Renal Medicine at Bristol Medical College.

Gene therapy – a technique that replaces or alters a defective 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 kidney disease.

The Purespring platform approach enables streamlined gene therapy development for kidney disease, offering the potential to halt, reverse and even cure rare and common kidney diseases.

The oversubscribed Series B funding round was led by Sofinnova Partners, in collaboration with a prominent syndicate including Gilde Healthcare, Forbion, British Patient Capital and founding investor Syncona Limited.

Proceeds will be used to support Purespring’s pipeline, including the initiation of a Phase I/II clinical trial for IgA Nephropathy (IgAN), a common chronic kidney disease that primarily affects young adults.

About a third of patients with IgAN will lose kidney function within five years and require a kidney transplant or dialysis.

Professor Moin Saleem said: “This is world-leading work, co-led by Professor Gavin Welsh and Dr Wen Ding, which has emerged from 25 years of work and expertise developed at Bristol Renal in podocyte biology and glomerular disease.

“The potential here is to generate curative therapies for kidney diseases with huge unmet needs, and this investment will allow us to reach clinical trials in the two main indications. We look forward to progressing the gene therapy platform we have built to address other glomerular diseases that result from podocyte damage and continue to build new treatments for even more clinical indications in the renal field.”

Professor Jeremy Tavare, Pro-Vice-Chancellor of Health and Life Sciences at the University of Bristol, said: “The significant progress made by Purespring in translating UoB’s groundbreaking research into renal gene therapy has been recognized by a high-profile investment syndicate. caliber. Their experience and resources will enable the company to continue its impressive trajectory and enable the clinical development of much-needed therapies for patients suffering from chronic kidney disease.”

Julian Hanak, CEO of Purespring, added: “Almost a tenth of the world’s population, around 840 million people, suffer from chronic kidney disease. For many of them, there are few options other than dialysis and transplantation. Our new treatment platform and deep understanding of kidney disease put us in a position to stop, reverse and even cure kidney disease. The funds raised will allow us to bring our new treatments to patients at the clinic.

“I am delighted to be able to welcome a syndicate of high-quality investors to support Purespring on this journey. Having sophisticated, experienced and committed investors on board who understand drug development and the urgency of bringing these much-needed therapies to patients will maximize opportunities for the company and the patients we treat.”

The funding follows critical scientific milestones for Purespring. Earlier this year, Purespring presented preclinical data at the 61st Congress of the European Renal Association (ERA) establishing the potential of AAV gene therapy to deliver transgenes to the podocyte to replace defective genes or to modulate protein production, supporting the potential of the AAV as an important novel. modality to treat a wide range of kidney diseases.

In 2023, a paper published in Science Translational Medicine demonstrated that Purespring successfully used AAV gene therapy to transduce the podocyte in vivo for the first time – an important milestone in overcoming obstacles that previously limited development in this area.

Purespring will publicly present its IgAN data for the first time at the American Society of Nephrology (ASN) Kidney Week conference on October 26 in San Diego.

Maina Bhaman, partner at Sofinnova Partners, said: “Purespring Therapeutics is a pioneer in the treatment of kidney diseases. The team’s ambition and caliber epitomize Sofinnova’s mission of scientific and business excellence in delivering transformative treatments to patients. We are excited to support the Purespring team as they bring their game-changing innovation to the global stage.”

Michael Kyriakides, investment partner at Syncona Investment Management and director of the board of Purespring Therapeutics, said: “Synona founded Purespring with the vision of creating the world’s leading renal gene therapy company and we have been impressed by its progress.

“The current financing and strong syndicate of investors the Company has attracted expands Purespring’s financial scale and clearly validates its technology, strategy and execution to date. We look forward to working alongside Purespring and its new syndicate as it prepares for its Phase I/II clinical trial.”