One-Time gene fix could ease PKU's strict diet burden
Disease control
Recruiting now
This early-stage trial tests a gene therapy called NGGT002 for adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The treatment delivers a working copy of the missing gene via a single IV infusion. Researchers will mon…
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC