One-Shot gene therapy could ease PKU diet burden
Disease control
Not yet recruiting
This early-stage study tests a single injection of a gene therapy (GS1168) in 9 adults with classical PKU whose condition is not well controlled by diet or other treatments. The goal is to see if the therapy is safe and can lower harmful phenylalanine levels in the blood, potenti…
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated May 12, 2026 13:39 UTC