Gene therapy before birth: a new hope for babies with GM1 disease
NCT ID NCT07479953
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests giving a gene therapy to unborn babies diagnosed with GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The therapy uses a harmless virus to deliver a working copy of the missing enzyme gene. The main goal is to see if the procedure is safe for both mother and baby, with only 5 participants planned.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AAV9 vector carrying the human beta-galactosidase gene
What this could lead to
If it works, this could point toward a way to treat GM1 gangliosidosis before birth, potentially slowing or preventing severe brain damage.
What could go wrong
This is a very early, small trial with only 5 participants. It is focused on safety, not yet on effectiveness. There are risks to both mother and fetus, including serious side effects or death.
Disclaimer
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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.
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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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.