GS1168

GS1168 is an adeno-associated virus (AAV) vector-based gene therapy developed by Gritgen Therapeutics for the treatment of phenylketonuria (PKU). It is designed to deliver a functional copy of the human phenylalanine hydroxylase (PAH) gene to the liver. PKU is a rare metabolic disorder caused by mutations in the PAH gene, leading to the accumulation of phenylalanine in the blood, which can cause severe neurological damage if untreated. By restoring PAH enzyme activity, GS1168 aims to normalize blood phenylalanine levels and reduce the need for strict dietary restrictions. It is currently being evaluated in clinical trials for adult patients with classical PKU.

Other names

GS1168 injectionGS-1168 injectionGS 1168 injectionAAV-PAH gene therapy