PS-002

PS-002 is an investigational gene therapy developed by Purespring Therapeutics for the treatment of primary IgA nephropathy (IgAN), a chronic autoimmune kidney disease. It utilizes an adeno-associated viral (AAV) vector to deliver therapeutic genetic material specifically to podocytes, the specialized cells in the glomeruli of the kidney. By targeting podocytes, PS-002 aims to modulate complement activation and control complement signaling exclusively within the glomeruli, addressing a key driver of disease progression in IgAN. Preclinical studies have demonstrated that administration of PS-002 reduces signs of kidney dysfunction, lowers complement deposition, and ameliorates scarring and structural damage in animal models. The therapy has received orphan drug designation from the European Medicines Agency (EMA) for IgA nephropathy and is progressing toward Phase I/II clinical trials[3][4][5][7][9].