One-Shot gene therapy could free PKU patients from strict diet
Disease control
Not yet recruiting
This early-phase trial tests a single injection of a gene therapy called GS1168 in 9 adults with PKU whose condition is not well controlled by diet or other treatments. The therapy uses a harmless virus to deliver a working copy of the PAH gene, which is faulty in PKU, to help th…
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC