Rewriting the code: a One-Time gene fix for a rare immune disease?
NCT ID NCT07775313
First seen Aug 20, 2026 · Last updated Aug 21, 2026 · Updated 1 time
Summary
This trial tests a one-time gene therapy for WHIM syndrome, a rare inherited immune disorder that causes severe infections. Doctors collect a person's own blood stem cells, use base editing to correct the faulty CXCR4 gene, and return the edited cells to the body. The goal is to see if this approach is safe and can restore a working immune system, potentially curing the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Base-edited autologous hematopoietic stem and progenitor cells
- What this could lead to
- If successful, this one-time gene therapy could provide a lasting cure for WHIM syndrome, eliminating the need for ongoing infection management.
- What could go wrong
- This is an early-stage trial with only 10 participants, so results may not generalize. The conditioning regimen carries risks like infection and organ damage, and the gene editing may not work in all cells.
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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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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
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