Autosomal dominant polycystic kidney disease inhibitors

Autosomal dominant polycystic kidney disease (ADPKD) is a progressive monogenic disorder characterized by growth of renal cysts, which eventually impair kidney function and the most common inherited cause of end-stage kidney disease worldwide, affecting approximately 500,000 people in the United States alone. Current therapeutic options remain limited, non-curative, and prone to safety concerns, underscoring a critical need for innovation. In collaboration with IRCCS Ospedale San Raffaele, CD3 is developing small molecules blocking cystic expansion, offering a transformative, orally bioavailable therapeutic strategy to stabilize renal function and improve patient outcomes.