Groundbreaking gene therapy aims to fix immune system in rare disease
NCT ID NCT06559176
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a new gene therapy (PM359) for people with a rare immune disorder called chronic granulomatous disease (CGD). The treatment takes a patient's own blood stem cells, fixes a specific genetic error using a precise editing tool called prime editing, and returns the corrected cells to the patient. The goal is to restore the immune system's ability to fight infections, but ongoing monitoring is needed. The study enrolls 12 participants and follows them for 15 years.
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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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Locations
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CHU - Sainte Justine Hospital
Montreal, Quebec, H3T 1C5, Canada
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NIH Clinical Center
Bethesda, Maryland, 20892, United States
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The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, 37203, United States
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University College of London Hospital
London, England, NW1 2PG, United Kingdom
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University of California Los Angeles Medical Center
Los Angeles, California, 90027, United States