One-Dose gene therapy could change life for hemophilia patients

NCT ID NCT07548411

First seen Apr 25, 2026 · Last updated May 08, 2026 · Updated 2 times

Summary

This study tests a single-dose gene therapy for Chinese adults with severe hemophilia A, a bleeding disorder. The treatment uses a harmless virus to deliver a gene that helps the liver produce a missing clotting protein. Seven participants are being monitored for safety and how well the therapy works.

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Contacts and locations

Locations

  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

    Tianjin, Tianjin Municipality, 300020, China

  • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

    Wuhan, Hubei, 430022, China

Conditions

Explore the condition pages connected to this study.