TQB-001

TQB-001 is an adeno-associated virus (AAV) gene therapy candidate developed by Torque Bio for the treatment of Autosomal Dominant Polycystic Kidney Disease type 2 (ADPKD2). The therapy utilizes a novel AAVk.20 capsid to deliver a functional copy of the PKD2 gene, which encodes the polycystin-2 (PC2) protein. ADPKD2 is a genetic disorder characterized by the formation of fluid-filled cysts in the kidneys, leading to progressive renal failure, and is caused by mutations in the PKD2 gene. TQB-001 aims to restore polycystin-2 function through gene replacement, potentially slowing or halting disease progression. The program is currently in the preclinical stage, with IND-enabling activities underway.