One-Shot gene therapy tested for rare metabolic disorder
Disease control
Not yet recruiting
This early-stage study is testing a single injection of an experimental gene therapy called GS1168 in adults with a genetic condition called PKU. The goal is to see if the treatment is safe and if it can help the body better process protein, potentially reducing the need for a st…
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC