uniQure hopeful in gene therapy trial
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Gene TherapyNetherlands

uniQure hopeful in gene therapy trial

08.01.2016 - A new hope for gene therapy: Dutch biotech uniQure has presented promising results from its haemophilia B gene therapy clinical trial. A jump in the company’s shares followed the announcement.

In a low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients, uniQures AAV5/FIX gene therapy, AMT-060, has resulted in encouraging data. Topline results showed that four out of the five patients were able to discontinue prophylactic recombinant factor IX. Two patients’ factor IX rose to 5.5% and 4.5% of the normal level, from below 2% before the treatment.

Haemophilia B patients themselves cannot produce enough factor IX, a protein responsible for blood clotting, leading to excessive bleeding. Patients are currently treated by repeated infusion of recombinant factor IX. uniQure’s gene therapy AMT-060 consists of a codon-optimised wild type FIX gene and the LP1 liver promoter together with the AAV5 viral vector, manufactured using uniQure’s insect cell based manufacturing technology. AMT-060 is administered, without immunosuppressant therapy, through the peripheral vein in a one treatment session for approximately 30 minutes.

“These early data from the low-dose cohort suggest that AMT-060 is generally well-tolerated and capable of successfully transducing the liver resulting in clinically meaningful FIX expression levels,” uniQure said in a statement. Frank W.G. Leebeek an investigator in the study at the Erasmus Medical Center in Rotterdam, added: “Thus far, the overall tolerability and FIX expression profile in the low-dose cohort is encouraging for patients with hemophilia B and support  the continuation of the study.”

Shareholders shared the company’s optimism. uniQure’s shares rose from below US$15 on 6th January, to more than US$17 on the 8th.

“Today, we are the only AAV gene therapy company in the world with both proprietary, commercial-scale manufacturing capabilities and encouraging clinical data across multiple diseases,” commented uniQure CEO Dan Soland.


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No liability assumed, Date: 29.06.2016