Purespring Therapeutics Unveils Preclinical Data on IgA Nephropathy Gene Therapy at ASN Kidney Week 2024

Purespring Therapeutics, a pioneering gene therapy company focused on kidney disease, has showcased promising preclinical data at the American Society of Nephrology (ASN) Kidney Week 2024 in San Diego, USA. The data reveals the significant potential of Purespring’s lead AAV gene therapy program, PS-002, in treating IgA nephropathy (IgAN), an autoimmune kidney disease. The presentation highlighted the ability to effectively target transgenes to podocytes, thereby modulating complement activation and reducing indicators of kidney disease in animal models.

The PS-002 therapy employs a modified virus to treat podocytes. Research demonstrated that administering PS-002 to a mouse model of IgAN reduced kidney dysfunction, lowered complement deposition, and improved structural characteristics of kidney disease. In pig models, the treatment resulted in elevated and prolonged gene expression in kidney tissues without any safety concerns.

Ambra Cappelletto, Principal Scientist at Purespring, emphasized that targeting podocytes to modulate complement activation is a highly effective therapeutic strategy. She highlighted that PS-002 could become the first gene therapy developed for IgA nephropathy, showing the efficiency of delivering therapeutic genetic material to podocytes. This opens up new possibilities for treating a broad range of kidney diseases.

Julian Hanak, Chief Executive Officer of Purespring, discussed the critical unmet need in chronic kidney disease, which affects approximately 840 million people worldwide. He expressed excitement about presenting their novel platform technology to the international nephrology community. Hanak pointed out that the data from ASN Kidney Week 2024 underscores the potential of Purespring’s gene therapy platform to deliver efficient and specific gene copies directly to podocytes. As the development of PS-002 progresses, there is hope that these promising results will translate into significant clinical benefits for patients.

Purespring recently secured £80 million ($105 million) in Series B financing to support the initiation of a Phase I/II clinical trial for IgAN. Currently, there is no cure for IgAN, and about one-third of patients experience kidney function loss within five years, necessitating kidney transplants or dialysis. Purespring is the first company to successfully treat kidney disease models by targeting podocytes through its proprietary adeno-associated virus (AAV) gene therapy platform.

The data presented at ASN follows previous critical preclinical data presented at the 61st European Renal Association (ERA) Congress in May. The earlier data established the potential of AAV gene therapy to deliver transgenes to podocytes, replacing defective genes or modulating protein production.

Purespring Therapeutics was founded on the work of Professor Moin Saleem from the University of Bristol. The company is committed to developing gene therapies to halt or prevent kidney disease, one of the most poorly treated disease areas. Purespring’s platform approach streamlines gene therapy development for both monogenic and non-monogenic renal diseases, offering the potential to halt, reverse, and even cure rare and common kidney diseases.

The company has a robust pipeline, including the lead asset for treating IgAN and other complement-mediated kidney diseases. They also have programs targeting diseases caused by mutations in the NPHS2 gene and other monogenic glomerular kidney diseases. Based in London, Purespring’s team combines expertise in podocyte biology and kidney disease with extensive experience in gene therapies, fostering a culture of diversity, creativity, and delivery. The company is supported by leading biotech investors and has raised £115 million to date.