Novel Gene Therapy Shows Promise for Treating IgA Nephropathy, Purespring Therapeutics Reports

Purespring Therapeutics presented encouraging preclinical data on its lead gene therapy candidate, PS-002, for the treatment of IgA nephropathy (IgAN), an autoimmune kidney disease. Findings shared at the American Society of Nephrology (ASN) Kidney Week 2024 highlighted PS-002’s potential to modulate disease progression by specifically targeting podocytes and reducing complement activation, marking a significant step forward in gene therapy for kidney disease.

Preclinical Success in Mouse and Pig Models

Purespring’s PS-002 uses an adeno-associated virus (AAV) to deliver therapeutic genes directly to podocytes—specialized kidney cells implicated in an estimated 60% of renal diseases, including IgAN. In preclinical testing, the administration of PS-002 in a mouse model of IgAN showed reduced kidney dysfunction, lowered complement deposition, and minimized kidney scarring, indicating improvement in kidney structure. Testing in pigs further demonstrated elevated and sustained gene expression in kidney tissue without notable safety issues, suggesting PS-002 may be both effective and well-tolerated.

Addressing an Unmet Need in Kidney Disease

With no cure currently available for IgAN, a condition that can lead to kidney failure, patients often face the need for dialysis or a transplant in advanced stages. Purespring’s novel AAV-based platform aims to provide durable gene therapy options by delivering precise genetic material to podocytes, potentially offering a long-lasting solution for patients. This approach aligns with the significant need for innovative treatments in chronic kidney disease, an area affecting hundreds of millions globally.

Backed by recent investor funding, Purespring plans to advance PS-002 into Phase I/II clinical trials. If successful, this program could represent a transformative option for IgAN, potentially altering disease progression in a manner not previously achievable with current therapies.