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The following is an excerpt from a press release from uniQure.


uniQure announced that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). “As a one-time administered therapy, AMT-061 has the potential to transform the treatment paradigm for hemophilia B patients,” said Annette von Drygalski, M.D., Associate Clinical Professor at the University of California San Diego and Director of its Hemophilia and Thrombosis Treatment Center, where the patient was treated. We hope to complete patient enrollment shortly and provide initial data before the end of the year.

Patient enrollment is also underway in the global Phase 3 HOPE-B clinical trial to evaluate the safety and efficacy of AMT-061.  Approximately 50 adult hemophilia B patients classified as severe and moderately-severe will be enrolled in a six-month observational period during which time they will continue to use their current standard of care to establish a baseline control.  After the six-month lead-in period, patients will go onto receive a single intravenous administration of AMT-061.  Treatment of patients in the HOPE-B pivotal trial is expected to start in the first quarter of 2019. “AMT-061 has the potential to be a major advancement in gene therapy for patients affected by hemophilia B,” stated Steven Pipe, M.D., Professor of Pediatrics and Pathology and Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program at the University of Michigan and principal investigator of the HOPE-B clinical trial.  “A one-time treatment, such as AMT-061, could be life-changing for these patients, many of whom struggle to manage ongoing challenges, including compliance with frequent infusions and recurrent episodes of bleeding.”

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