Gene therapy trial aims to help people with angelman syndrome communicate better
NCT ID NCT07181837
First seen Jun 26, 2026 · Last updated Jul 24, 2026 · Updated 2 times
Summary
This early-phase trial tests a gene therapy called MVX-220 in 12 children and adults with Angelman syndrome. The therapy delivers a working copy of the UBE3A gene via a single injection into the fluid around the brain. Researchers are primarily checking safety, but also looking for improvements in communication skills using a caregiver-reported measure.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- MVX-220 gene therapy (a modified virus carrying a working UBE3A gene)
- What this could lead to
- If it works, this could point toward a treatment that improves communication and daily function for people with Angelman syndrome.
- What could go wrong
- This is a very early, small trial with only 12 participants, so results may not apply to everyone. Gene therapy also carries risks like immune reactions or side effects from the injection.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cedars-Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Other studies related to the condition(s) this trial covers.
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