One-Shot gene therapy aims to fix rare genetic disorder
⭐️ CURE ⭐️
Recruiting now
This early-stage study is testing a new one-time gene therapy called NGGT002 for adults with classic phenylketonuria (PKU). The goal is to see if delivering a working copy of the faulty gene is safe and can lower harmful blood levels of phenylalanine, potentially reducing the nee…
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: ⭐️ CURE ⭐️
Last updated Mar 13, 2026 15:05 UTC