Gene therapy injection tested for rare nerve disease

NCT ID NCT05361031

Completed Disease control Sponsor: Helixmith Co., Ltd. Source: ClinicalTrials.gov ↗

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.

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.

Charcot-Marie-Tooth disease Charcot-Marie-Tooth disease type 1A

As listed by the trial registrant

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

CMT

Contacts and locations

Locations

Samsung Medical Center, Seoul National University Hospital

Seoul, 06351, South Korea

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