AAV gene therapy for ADPKD

AAV gene therapy for ADPKD refers to a therapeutic approach, notably being developed by Torque Bio, that utilizes adeno-associated virus (AAV) vectors to deliver functional copies of the PKD1 or PKD2 genes to the kidney. This gene replacement strategy aims to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD) by restoring the function of polycystin proteins, which are deficient or defective in patients with the condition. The Torque Bio program employs novel, engineered AAV capsids optimized for kidney transduction and utilizes a local kidney administration route to maximize delivery to target cells—including proximal tubules, distal tubules, and collecting ducts—while minimizing systemic exposure and off-target effects in the liver. Preclinical studies in mouse models have demonstrated that this approach can rescue cystic phenotypes, significantly reducing kidney weight, cystic index, and blood urea nitrogen (BUN) levels, while improving glomerular filtration rate (GFR).