Hemophilia A-Factor VIII - Healing Genes

Hemophilia A-Factor VIII

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Factor VIII Gene Therapy Study in Patients With Hemophilia A

A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A

Phase 1 / 2


Doctors at five research hospitals seek male patients with Hemophilia A (with Factor VIII Inhibitors) to trial a new product, BAY2599023, an investigatory gene therapy. This therapy utilizes a harmless virus to deliver a functional copy of the F8 gene which may permanently provide sufficient blood coagulation and reduce the need for transfusions.

The treatment requires a single intravenous injection of BAY2599023 and follow up will involve lab assessments and check-ins over the following year and for up to 5 years.


  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Not have participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks


  1. Screening before the treatment
  2. A single infusion of the gene therapy drug will be administered
  3. Monitoring of the patient for a few hours after dosing, with periodic lab assessments required for 52 weeks and up to 5 years..

Trials will take place at five locations:
Arkansas Children’s Hospital in Little Rock, AK, Map.
Loma Linda Children’s Hospital in San Bernardino, CA, Map.
C.S. Mott Children’s Hospital in Ann Arbor, MI, Map.
University Hospitals Cleveland Medical Center in Cleveland, OH, Map.
University of Wisconsin in Madison, WI, Map.
Bayer Clinical Trials Contact  |  (+) 1-888-8422937 |  [email protected]
For trial location information (Phone Menu Options ‘3’ or ‘4’) (+)1-888-842-2937
Ultragenyx Pharmaceutical Inc

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