Gene Therapy Road Map

Gene therapy for hemophilia involves using a modified virus to deliver a working copy of a dysfunctional gene. Sufferers of hemophilia lack factors that allow for normal clotting. The copy they receive from gene therapy encodes these essential clotting factors, allowing clotting to proceed normally.

How Gene Therapy Can Help Hemophilia

Gene therapy research offers the hope of a one-time cure for those with hemophilia. Clinical trials are offering promising results, as many participants have been able to sustain high levels of critical clotting factors.

The Gene Therapy Roadmap

Patients often worry about the safety and efficacy (effectiveness) of the treatment. However, studies show that nearly 90 percent of trial participants see normal or near-normal levels of anticoagulant factor activity.

Gene transfer occurs using a non-pathogenic and replication-deficient virus, so the safety concerns are minimal. However, there is always a risk of infusion reactions from the experimental gene therapy, as well as side effects of immunosuppression therapy, if it is needed to reduce elevations in liver enzymes.

It is important to also know that gene therapy is only effective in treating the patient. It does not correct the mutated gene that causes hemophilia or prevent a parent from passing that gene to their child.

The International Society on Thrombosis and Haemostasis (ISTH) consists of world-renowned experts in the global hemophilia community. In addition, ISTHs develops education resources that provide patients and families with a front-row seat in the development of this amazing new treatment protocol.

ISTH is helping patients understand gene therapy and how it may one day be a cure for a condition that currently requires a lifetime of treatment.


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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., and Spark Therapeutics

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