Experimental gene 'Silencer' therapy enters human testing for rare muscle disease
NCT ID NCT06907875
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows any signs of working. Participants receive a single IV dose and are monitored for about 5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
EPI-321 (a gene therapy that aims to silence the faulty gene causing FSHD)
What this could lead to
If it works, this could point toward a treatment that stops or slows muscle damage in FSHD, a rare muscular dystrophy.
What could go wrong
This is a very early, small trial (12 people) focused on safety, not proof of effectiveness. The therapy is new and may not work or could cause side effects.
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.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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David Geffen School of Medicine at University of California, Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Kennedy Krieger Institute, Center for Genetic Muscle Disorders
RECRUITINGBaltimore, Maryland, 21205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Pacific Clinical Research Network
RECRUITINGAuckland, 0622, New Zealand
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 303329, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Royal Alfred Hospital
RECRUITINGSydney, New South Wales, 2050, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Massachusetts Chan Medical School
RECRUITINGWorcester, Massachusetts, 01605, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Utah Program for Inherited Neuromuscular Disorders - University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••