Gene fix for rare immune disease: can it stop deadly infections?

NCT ID NCT03645486

Recruiting now Disease control Sponsor: Shenzhen Geno-Immune Medical Institute Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This trial tests a gene therapy for chronic granulomatous disease (CGD), a rare immune disorder that leaves patients vulnerable to severe infections. Ten patients will receive their own stem cells modified with a lentivirus to correct the faulty gene. The goal is to restore immune function and reduce infection frequency, with safety monitored for 15 years.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: lentiviral gene therapy (TYF-CGD-modified stem cells)
What this could lead to: If successful, this could provide a long-term way to control CGD by fixing the faulty gene, reducing severe infections and hospitalizations.
What could go wrong: This is a very early, small trial (10 people) testing safety and effectiveness. Gene therapy carries risks like immune reactions or the treatment not working as expected.

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.

chronic granulomatous disease

As listed by the trial registrant

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

CHRONIC GRANULOMATOUS DISEASE

Contacts and locations

Locations

Shenzhen Geno-immune Medical Institute
RECRUITING

Shenzhen, Guangdong, 518000, China

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