Gene therapy heals wounds in rare skin disease?
NCT ID NCT04213261
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests a gene therapy called FCX-007 for people with recessive dystrophic epidermolysis bullosa, a rare disease that causes fragile skin and painful blisters. The therapy uses the patient's own skin cells, genetically corrected to produce collagen, and injects them into wounds. The study compares treated wounds to untreated ones in just 6 participants, looking for complete healing by 24 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- FCX-007 (dabocemagene autoficel) – a gene-corrected skin cell therapy
- What this could lead to
- If it works, this could provide a way to heal chronic wounds in people with a severe skin blistering disease, improving quality of life.
- What could go wrong
- This is a very small early-phase trial with only 6 people, so results may not apply to everyone. The treatment involves injections and there is a risk of immune reactions or the wounds not healing.
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
Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Dell Children's Medical Group
Austin, Texas, 78723, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Solutions Through Advanced Research, Inc.
Jacksonville, Florida, 32256, United States
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Stanford University
Stanford, California, 94305, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy for 'Butterfly Skin' disease tracked for 15 years in major safety registry
- Anti-Inflammatory drug may ease swallowing in rare skin disease
- New hope for blistering skin diseases: drug targets antibodies to heal wounds
- Stanford launches study to better understand rare blistering skin disease
- Gene therapy gel aims to stop skin cancer from coming back in RDEB patients
- New cream could help kids with 'Butterfly Skin' heal faster