Purespring Therapeutics

Purespring Therapeutics is a London-based biotechnology company established in 2020 by Syncona, a life science investor, and built upon the research of founders Professor Moin Saleem and Mauro Giacca. The company is a pioneer in developing adeno-associated virus (AAV) gene therapies specifically for chronic kidney diseases, an area with significant unmet medical needs. Purespring's scientific foundation is the work of Professor Moin Saleem of the University of Bristol, a leader in pediatric renal medicine whose research has focused on the podocyte—a specialized kidney cell critical to filtration and implicated in approximately 60% of renal diseases.

The company's proprietary platform is engineered to deliver gene therapies directly to podocytes, offering the potential to halt or even reverse kidney disease by correcting defective genes or modulating protein production. This targeted approach aims to provide curative treatments for both rare monogenic and more common kidney conditions, moving beyond options like dialysis or transplantation which only manage late-stage disease. Purespring's business model is centered on the research, development, and eventual commercialization of these novel therapies. Revenue generation is anticipated through product sales and strategic partnerships within the pharmaceutical and healthcare sectors.

Purespring has secured significant funding to advance its pipeline, raising approximately $60 million in a Series A round in 2020 and $105 million in a Series B round in October 2024. This capital is being used to progress its lead candidate, PS-002 for IgA Nephropathy (IgAN), into a Phase I/II clinical trial. The company's pipeline also includes programs for other conditions, such as steroid-resistant nephrotic syndrome. Preclinical data has demonstrated the platform's ability to successfully deliver genetic material to podocytes in animal models, restoring kidney function and showing no off-target effects or safety concerns.

Keywords: gene therapy, kidney disease, AAV platform, podocyte targeting, renal disease, nephrology, IgA Nephropathy, monogenic diseases, chronic kidney disease, therapeutics development, Moin Saleem, Syncona, adeno-associated virus, clinical trials, biopharmaceutical, life sciences, nephrotic syndrome, glomerular diseases, rare diseases, biotechnology