Gene therapy aims to fix bone marrow failure in fanconi anemia

NCT ID NCT03351868

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study evaluates safety and whether the treatment can improve blood cell counts and reduce disease complications.

What this could mean

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

Active substance: gene-modified stem cells
What this could lead to: If successful, this gene therapy could restore normal blood cell production and reduce the need for transfusions or bone marrow transplants in people with Fanconi anemia.
What could go wrong: This is an early-phase trial with only 10 participants, so results may not apply to everyone. Risks include immune reactions or the gene therapy not working as expected.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for FANCONI ANEMIA are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Fanconi anemia Fanconi anemia complementation group A

As listed by the trial registrant

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

FANCONI ANEMIA

Contacts and locations

Locations

Shenzhen Geno-immune Medical Institute
RECRUITING

Shenzhen, Guangdong, 518000, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.