Gene therapy gel targets rare blistering skin disease
NCT ID NCT07717346
First seen Jul 21, 2026 · Last updated Jul 22, 2026 · Updated 1 time
Summary
This early study tests a new gene therapy gel called KB111 for Hailey-Hailey disease, a rare genetic condition that causes painful blisters and skin lesions. The gel uses a modified virus to deliver a working copy of the faulty gene directly to the skin. The trial enrolls about 7 people aged 12 and older to see if the treatment is safe and whether it can reduce symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- a topical gel containing a modified herpes simplex virus that delivers a working copy of the ATP2C1 gene
- What this could lead to
- If successful, this could point toward a treatment that reduces painful blisters and improves quality of life for people with Hailey-Hailey disease.
- What could go wrong
- This is a very early, small study (7 people) focused mainly on safety. The treatment may not work or could cause side effects, and larger trials are needed.
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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Pediatric Skin Research
RECRUITINGMiami, Florida, 33156, United States
Contact Phone: •••-•••-••••
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