PS-001

PS-001 is an investigational gene therapy developed by Purespring Therapeutics for the treatment of steroid-resistant nephrotic syndrome caused by mutations in the NPHS2 gene (encoding podocin). It utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of NPHS2 specifically to kidney podocytes. The therapy is designed to restore podocin expression and function in glomerular diseases, with preclinical data demonstrating effective and specific transgene delivery and expression in kidney podocytes. This approach aims to stop, reverse, or potentially cure chronic kidney diseases associated with defective podocin[4][2]. As of May 2025, it is the first known AAV-based gene therapy targeting podocytes for this indication.