Purespring Therapeutics has secured $105 million (£80m) in a Series B funding round to initiate Phase I/II clinical trials for PS-002, an experimental adeno-associated viral (AAV) gene therapy targeting IgA nephropathy (IgAN). The funding, led by Sofinnova Partners, will support the advancement of this novel treatment designed to address the underlying causes of IgAN, a significant cause of end-stage renal disease.
Targeting Podocytes in IgAN
IgAN is an autoimmune kidney disease characterized by the accumulation of immunoglobulin A (IgA) protein in the kidneys, leading to inflammation and potential kidney failure. Purespring's PS-002 is designed to target podocytes, specialized cells crucial for kidney function. In IgAN, these cells are damaged by immune complexes, resulting in protein leakage and kidney dysfunction. By delivering therapeutic genes directly to podocytes, PS-002 aims to restore normal kidney function and prevent disease progression.
Preclinical Data and Platform Technology
Purespring presented preclinical data for its PS-001 therapy at the European Renal Association (ERA) Congress in May 2024, demonstrating the ability of AAV gene therapy to efficiently target podocytes. This approach allows for the replacement of defective genes or the modulation of protein production within the kidney. The company's proprietary 'gene search engine' platform, FunSel, facilitates the selection of optimal treatments from a diverse library of AAV vectors, enhancing the precision and efficacy of their gene therapies.
Investment and Market Context
The Series B funding round was led by Sofinnova Partners, with participation from Gilde Healthcare, Forbion, British Patient Capital, and founding investor Syncona. This investment reflects growing interest in the IgAN therapeutic landscape, highlighted by recent acquisitions such as Novartis' $3.2 billion purchase of Chinook Therapeutics, gaining access to atrasentan and zigakibart, both in Phase III trials for IgAN.
Addressing Unmet Needs in Kidney Disease
According to Purespring’s CEO Julian Hanak, approximately 840 million people worldwide suffer from chronic kidney disease, with limited options beyond dialysis and transplantation for many. Purespring's therapeutic platform aims to halt, reverse, and potentially cure kidney disease by leveraging a deep understanding of kidney biology and innovative gene therapy approaches. The Phase I/II trial of PS-002 represents a crucial step towards providing new treatment options for patients with IgAN and other kidney disorders.
