One-Shot gene therapy could free PKU patients from strict diet

NCT ID NCT07318909

Not yet recruiting Disease control Sponsor: Gritgen Therapeutics Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tests a single injection of a gene therapy called GS1168 in 9 adults with PKU whose condition is not well controlled by diet or other treatments. The therapy uses a harmless virus to deliver a working copy of the PAH gene, which is faulty in PKU, to help the body break down phenylalanine. The main goal is to check safety, but researchers will also measure changes in blood Phe levels and quality of life.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: GS1168 injection (a gene therapy using a harmless virus to deliver a working PAH gene)
What this could lead to: If successful, this could point toward a one-time treatment that helps people with PKU control their phenylalanine levels without strict diet or daily medication.
What could go wrong: This is a very early, small study (9 people) focused on safety. The gene therapy may not work well enough, could cause side effects, or may not lead to a lasting benefit.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHENYLKETONURIA (PKU)

Contacts and locations

Locations

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, 230022, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.