One-Shot gene therapy could free hemophilia b patients from frequent injections

NCT ID NCT05203679

Ongoing Disease control Sponsor: Shanghai Xinzhi BioMed Co., Ltd. Source: ClinicalTrials.gov ↗

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

Summary

This clinical trial is testing a one-time gene therapy called BBM-H901 for people with hemophilia B, a bleeding disorder caused by low levels of clotting factor IX. The therapy uses a harmless virus to deliver a working copy of the factor IX gene to the liver, so the body can produce its own clotting factor. The study involves 32 adult men with severe hemophilia B and will monitor safety, liver function, and how well the treatment reduces bleeding episodes over time.

What this could mean

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

Active substance: BBM-H901 (a gene therapy using a harmless virus to deliver a working copy of the factor IX gene to the liver)
What this could lead to: If it works, this could give people with hemophilia B a lasting increase in clotting factor, reducing or eliminating the need for regular injections.
What could go wrong: This is an early-to-mid stage trial with only 32 participants. The gene therapy may not work for everyone, and there are risks like liver inflammation or an immune reaction that could reduce its effect.

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

Anhui Provincial Hospital

Hefei, Anhui, 230022, China
Henan Cancer Hospital

Zhengzhou, Henan, 450003, China
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, Tianjin Municipality, 300020, China
Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, 510515, China
North China University of Science and Technology Affiliated Hospital

Tangshan, Hebei, 063000, China
Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100005, China
The Second Hospital of Shanxi Medical University

Taiyuan, Shanxi, 030001, China
The Second People's Hospital of Shenzhen

Shenzhen, Guangdong, 518025, China
The second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650032, China

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