UniQure attempts to keep comeback on track with showcase

20 November 2018

Dutch biotech uniQure (Nasdaq: QURE), the company which failed with its attempts to market Glybera (alipogene tiparvovec), the world’s first approved gene therapy, could have consigned its troubles to the past through a successful rebuilding program.

Last week, the company’s shares jumped by more than a third in a single day after the firm announced positive data from a Phase IIb dose-confirmation study of AMT-061 in hemophilia B.

On Monday, the company followed this up by hosting an R&D day when it showed off its new pipeline of AAV gene therapies and their potential in hemophilia A, Fabry disease and spinocerebellar ataxia type 3. All have impressed in preclinical testing.

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