P-PAH-101

P-PAH-101 is a liver-directed gene therapy in preclinical development for the treatment of phenylketonuria (PKU), an inherited metabolic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene. PKU leads to a buildup of phenylalanine in the body, which can cause cognitive impairment if untreated. P-PAH-101 utilizes Poseida Therapeutics' proprietary Super piggyBac DNA delivery technology combined with a hybrid adeno-associated virus (AAV) and nanoparticle delivery system to deliver functional copies of the PAH gene to liver cells. Preclinical data have shown that this approach can normalize phenylalanine levels following a single treatment in animal models[3][7].