Groundbreaking gene therapy aims to fix immune system in rare disease

NCT ID NCT06559176

ENROLLING_BY_INVITATION Disease control Sponsor: Prime Medicine, Inc. Source: ClinicalTrials.gov ↗

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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Conditions

The condition(s) this trial relates to.

chronic granulomatous disease granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CHRONIC GRANULOMATOUS DISEASE GRANULOMATOUS DISEASE, CHRONIC

Contacts and locations

Locations

CHU - Sainte Justine Hospital

Montreal, Quebec, H3T 1C5, Canada
NIH Clinical Center

Bethesda, Maryland, 20892, United States
The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers

Nashville, Tennessee, 37203, United States
University College of London Hospital

London, England, NW1 2PG, United Kingdom
University of California Los Angeles Medical Center

Los Angeles, California, 90027, United States

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