Can a One-Time gene infusion rewrite the fate of PKU?
⭐️ CURE ⭐️
Recruiting now
This early-stage trial tests a single intravenous infusion of NGGT002, a gene therapy designed to deliver a working copy of the PAH gene to adults with classic phenylketonuria (PKU). The goal is to see if this approach can safely lower phenylalanine levels in the blood, which cou…
Phase: PHASE1, PHASE2 • Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. • Aim: ⭐️ CURE ⭐️
Last updated Aug 15, 2026 00:00 UTC