Purespring Therapeutics Receives UK Approval for PS-002 AAV Gene Therapy Trial in IgA Nephropathy

Purespring Therapeutics, a company focused on treating kidney diseases, has received UK regulatory approval for its planned Phase I/II study of PS-002, its lead AAV gene therapy program for IgA nephropathy (IgAN). The Clinical Trial Application (CTA) for this AAV therapeutic was approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA), the NHS Health Research Authority (HRA), and Research Ethics Committee (REC).

The approval follows a recent FDA Investigational New Drug (IND) clearance and EMA orphan drug designation, officially transitioning Purespring into a clinical-stage company. According to CEO Haseeb Ahmad, these regulatory milestones validate the company’s AAV approach of targeting kidney disease directly at its source, rather than just managing symptoms.

The AAV-delivered PS-002 is designed to modulate complement activation in the kidney by precisely targeting podocytes, which are key cells in the kidney’s filtering units. IgAN is a rare, chronic autoimmune disease where trapped protein causes inflammation and damage, often leading to kidney failure despite currently available therapies.

The multi-country Phase I/II trial for the AAV program is expected to enroll its first patient in the fourth quarter of 2025 across sites in the U.S. and Europe. The study will initially focus on evaluating safety and dose parameters, with the Phase 2 portion providing early markers of efficacy.