The WFH Gene Therapy Registry (GTR) is an online web-based system designed to collect data on people with hemophilia (PWH) who receive gene therapy as part of a clinical trial or as an approved product. This important initiative will enhance our understanding of gene therapy for hemophilia and ensure the bleeding disorders community has access to timely evidence on safety and efficacy parameters. The WFH is proud to announce that on October 8, 2024, the first patient was enrolled in the WFH GTR.
Gene therapy marks an important milestone in the development of treatment options for PWH, offering the potential to improve their clinical outcomes—including quality of life—with a one-time infusion. Due to the small number of PWH participants in the pivotal clinical trials and their relatively short nature, important questions on the long-term outcomes of gene therapy remain to be answered. The WFH Gene Therapy Registry (GTR) offers a solution for this by providing an invaluable resource to efficiently gather global data and gain valuable evidence on the potential long-term risks, safety, and effectiveness of gene therapy.
The enrollment of the first patient in the WFH GTR marks a significant milestone in ensuring the safety and efficacy of gene therapy for hemophilia patients worldwide. This global initiative will provide invaluable data to guide future treatments, and underscores our commitment to advancing treatment and improving the lives of people with bleeding disorders worldwide.
—Glenn Pierce, MD, PhD, Vice President, Medical, WFH
This first PWH enrolled in the WFH GTR marks the beginning of a large international effort by partner organizations, PWH, national registries and hemophilia treatment centres (HTCs) worldwide, to ensure all eligible PWH are invited to participate. Only through global efforts can we gather enough data on this rare disease population to inform policy and treatment decisions.
The WFH would like to thank the WFH GTR Steering Committee and the Scientific Advisory Board, co-chaired by Barbara Konkle, MD, and Flora Peyvandi, MD; partner organizations; regulatory agencies; national registries; HTCs and PWH who have been working together to make the WFH GTR a reality!
Partner organizations
| Industry organizations
| Countries Australia, Belgium, Brazil, Canada, France, Germany, Ireland, Italy, Japan, Korea, Netherlands, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Taiwan, U.K., U.S.A. |
To learn more about the WFH GTR, click here. To learn more about gene therapy, check out our GTR fact sheet.
The WFH Gene Therapy Registry is supported by WFH Founding visionary partners BioMarin Pharmaceutical Inc., CSL Behring, Pfizer, and Spark Therapeutics.