Could a One-Time gene fix end bleeding episodes for hemophilia b?

NCT ID NCT03961243

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 1 trial tests a gene therapy that uses a lentivirus to deliver a working clotting factor IX gene into patients' own stem cells. Ten males with severe hemophilia B will receive the modified cells. The goal is to see if it's safe and can raise clotting factor levels, potentially reducing bleeding episodes.

What this could mean

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

Active substance: Lentiviral factor IX gene modified stem cells
What this could lead to: If successful, this could allow people with hemophilia B to produce their own clotting factor, reducing or eliminating the need for regular factor IX infusions.
What could go wrong: This is an early Phase 1 trial with only 10 participants, so safety and effectiveness are not yet proven. There is a risk of side effects or the therapy 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.

hemophilia B

As listed by the trial registrant

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

HEMOPHILIA B

Contacts and locations

Locations

Shenzhen Geno-immune Medical Institute
RECRUITING

Shenzhen, Guangdong, 518000, China

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