Gene therapy injection tested for rare nerve disease
NCT ID NCT05361031
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Trial info
This early-phase trial tested the safety of a gene therapy called Engensis (VM202) in 12 adults with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder that causes muscle weakness. Participants received multiple injections into their leg muscles and were monitored for 270 days. The main goal was to check for side effects, not to measure how well the treatment works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Engensis (VM202) - a gene therapy given as injections into leg muscles
What this could lead to
If safe and effective, this could point toward a treatment that slows or improves muscle weakness in CMT1A.
What could go wrong
This is a very early, small trial (12 people) focused only on safety, not proof of benefit. The treatment may not work or could cause side effects.
Disclaimer
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the original study
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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.
Contacts and locations
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Locations
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Samsung Medical Center, Seoul National University Hospital
Seoul, 06351, South Korea