UniQure shares jump on promising gene therapy data

[January 07, 2016]  (Reuters) - Dutch biotech UniQure NV said on Thursday its experimental gene therapy for a blood disorder benefited some patients in an early-stage study, sending its shares up about 20 percent in light premarket trading.

The drug, AMT-060, is being developed to treat hemophilia B, a condition caused by the deficiency of the factor IX gene, which is instrumental in blood-clotting.

Gene therapy, a keenly watched area in drug development, aims to insert corrective genes into malfunctioning cells.

However, the field has been dogged by a series of disappointments and safety concerns in its more than 20 years of research.

UniQure said after being treated with the drug, four out of five patients were able to fully discontinue taking prophylactic rFIX, which is currently used as routine treatment for the condition.

One patient however, experienced an increase in liver enzyme levels, an indicator of liver damage, and was put on a short treatment course.

In the recent past, UniQure's rival Bluebird Bio also faced a setback when data on its drug, Lentiglobin, wasn't found to be as effective as expected.

Celladon Corp, another gene therapy developer, has suspended trial on a heart drug after it failed in a mid-stage study.

UniQure's shares were trading at $18 before the bell.

(Reporting by Amrutha Penumudi in Bengaluru; Editing by Saumyadeb Chakrabarty)

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