Gene therapy aims to help hemophilia a patients make their own clotting factor

NCT ID NCT03217032

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 early-stage trial tests a gene therapy for people with hemophilia A, a bleeding disorder. The treatment uses a modified virus to deliver a working gene into the patient's own stem cells, which are then infused back. The goal is to help the body produce clotting factor VIII, potentially reducing bleeding episodes. Only 10 participants are enrolled to check safety first.

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 VIII gene modified stem cells
What this could lead to: If successful, this could provide a long-term way for people with hemophilia A to produce their own clotting factor, reducing or eliminating the need for regular factor VIII infusions.
What could go wrong: This is a very early Phase 1 trial with only 10 participants, so safety and effectiveness are not yet proven. There is a risk of side effects, and the gene therapy may not work as hoped.

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 A

As listed by the trial registrant

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

HEMOPHILIA A

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Shenzhen Geno-immune Medical Institute
RECRUITING

Shenzhen, Guangdong, 518000, China

Contact Phone: •••-•••-•••• Email: •••••@•••••

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