One-Shot gene therapy could free PKU patients from strict diet
NCT ID NCT07318909
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.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PHENYLKETONURIA (PKU) are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230022, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••