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The First Affiliated Hospital Of Bengbu Medical University

Clinical trials sponsored by The First Affiliated Hospital Of Bengbu Medical University, explained in plain language.

Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1

New gene therapy aims to tame PKU in first human test

Disease control Recruiting now

This early-phase study tests a single IV dose of NGGT002 gene therapy in 15 adults with phenylketonuria (PKU), a genetic disorder that causes harmful buildup of phenylalanine. The main goal is to check safety over one year, with long-term follow-up for five years. Researchers wil…

Phase: EARLY_PHASE1 • Sponsor: The First Affiliated Hospital of Bengbu Medical University • Aim: Disease control

Last updated Jun 27, 2026 09:10 UTC