New gene therapy hopes to ease PKU's strict diet for life
Disease control
Recruiting now
This early-stage trial tests a one-time gene therapy called NGGT002 in 12 adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The therapy delivers a working copy of the PAH gene to help the body process phenylalanine no…
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated May 01, 2026 15:57 UTC