GENE THERAPY FOR HEMOPHILIA B

We have heard directly from people with hemophilia B that it’s important to them to lead a life free from the restrictions of their disease. Risk of bleeding and the burden of prophylactic replacement therapies, which often require multiple injections per week, can make that difficult.

In hemophilia B, there is a mistake in the gene that allows the body to make clotting factor IX. uniQure is working to develop a treatment for hemophilia B by using gene therapy to insert a working copy of the factor IX gene into patients to compensate for the genetic problem that causes this disease.

Let’s learn more about gene therapy

Understanding Gene Therapy: The Cookbook Metaphor

For the scientific explanation, watch this video

We are now studying AMT-061 in a phase 3 clinical trial called HOPE-B. 

AMT-061: the gene therapy used in HOPE-B
uniQure has an investigational gene therapy called AMT-061 currently in clinical trials for the treatment of hemophilia B. AMT-061 includes a working version of the gene for factor IX packaged into a viral shipping container that will deliver it to the liver. Once in the liver, this new factor IX gene will treat hemophilia by directing the cells of the liver to make factor IX.

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Things you may be wondering about gene therapy

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uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The company is developing several gene therapies for the treatment of patients with liver/metabolic, central nervous system, and other severe genetic diseases.

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