When we have talked about this trial with various people with hemophilia B, here were some of the questions they had…

If I take this gene therapy, will genetic changes be passed down to my children?
No. The AAV package used to carry the working copy of the factor IX gene does not integrate with your body's own DNA, so the gene will not be carried on to future generations of cells and is not transmitted to your future/potential offspring.

How long will the effects of AMT-061 last? Is AMT-061 a one-time treatment?
We don’t yet know how long the response to treatment with AMT-061 may last. We also do not know whether one or multiple infusions of AMT-061 may be necessary over time.

Will I still have to infuse factor IX?
Through clinical trials, we will determine whether AMT-061 can replace prophylactic factor IX therapy. However, if you experience a bleed, you will still be treated with factor IX.

Will the virus that carries the factor IX gene therapy make me sick?
The virus that carries AMT-061 is designed so that it cannot replicate and therefore cannot cause disease. As with any investigational therapy, evaluation of the safety of AMT-061 is a major component of this study.

Is AMT-061 safe?
AMT-061 has been administered to 3 patients with severe hemophilia; all of whom attained therapeutic levels of factor IX activity. None of these patients has thus far reported any bleeding events or required any infusions of factor IX replacement therapy. No serious adverse events have been reported.

In the ten patients who received AMT-060, 1 experienced a short fever after the administration of the drug, and 3 patients experienced elevations in liver enzymes. All adverse events were treated and resolved without recurrence. The ten patients who received AMT-060 have now been followed for up to 2.5 years without any additional occurrence of adverse events.

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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