Before etranacogene dezaparvovec, there was AMT-060.

AMT-060 was the previous gene therapy in development by uniQure to treat hemophilia B. It was studied in 10 patients, from whom we learned that with AMT-060 treatment factor IX activity was increased for at least 3.5 years, use of factor IX replacement therapy was decreased, and the frequency of bleeding events decreased.5

In the study of AMT-060, 4 patients experienced serious adverse events including 1 fever that resolved within 24 hours, and 3 increases in liver enzymes that resolved after standard treatment with a steroid medication. None of these events affected the ability of the medication to increase factor IX activity, and there have been no further treatment-related serious adverse events in the 3.5 years these patients have been followed.5,6

  • Reduction in Severity of Disease Still Evident 2.5 Years Later6

  • Reduction in Use of FIX Prophylaxis5,6

  • Reduction in Frequency of Spontaneous Bleeds Annually5

  • Click on image to enlarge

To further enhance the response to this gene therapy, we replaced the factor IX gene with a slightly modified form that was shown to produce significantly higher levels of factor IX activity compared to the form used in AMT-060. This new version of the gene therapy is called etranacogene dezaparvovec.

Etranacogene dezaparvovec has been studied in 1 trial to confirm that it causes the liver to produce higher levels of factor IX activity.7 No treatment-related serious adverse events have been reported for the 3 patients who received etranacogene dezaparvovec.* After receiving the gene therapy, 2 patients experienced brief incidents of increased liver enzymes (3 in one patient, 1 in another) considered unrelated to treatment, but these resolved without intervention or impact on FIX activity.7 Mean factor IX activity for the 3 patients was 45% when measured 36 weeks after treatment with etranacogene dezaparvovec.8 Etranacogene dezaparvovec is now being investigated in the HOPE-B phase 3 clinical trial.

*One patient underwent hip surgery due to a pre-existing condition and was treated perioperatively with short-acting factor replacement. This was reported by the investigator as a serious adverse event unrelated to etranacogene dezaparvovec.

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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