Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD, caused by the mutations of PKD1 or PKD2, is among the most common monogenetic disorders and a leading genetic cause of end-stage renal disease. The clinical hallmark of this disease is the presence of numerous renal tubule-derived cysts.  Excessive proliferation, a central pathological feature, fuels the relentless expansion of cysts, ultimately causing end-stage renal disease. ADPKD affects approximately 600,000 people in the US and 12.5 million people worldwide.


Regulus filed an investigational new drug (IND) package with the US Food and Drug Administration (FDA) for RGLS4326, targeting microRNA-17 (miR-17) for the treatment of autosomal dominant polycystic kidney disease (ADPKD).